Category: Market Access

  • FDA stifles Merck’s cough drug gefapixant, asking for more data

    The FDA has issued a complete response letter (CRL) to Merck & Co’s gefapixant candidate for refractory chronic cough, delaying the programme and giving Bayer an opportunity to close the gap with its rival eliapixant drug. Gefapixant is still out in front in the non-narcotic, orally administered selective P2X3 receptor antagonist class – having secured […]

  • After FDA setback, Ipsen gets first OK for rare disease drug palovarotene

    After regulatory hold-ups in the US and Europe, Ipsen has claimed a confidence-boosting approval in Canada for palovarotene, a rare disease therapy acquired via its $1.3 billion takeover of Clementia Pharma in 2019. Health Canada has given the drug a green light – under the Sohonos brand – to treat fibrodysplasia ossificans progressiva (FOP), making […]

  • Chronic kidney disease: treating a silent killer

    In November, NICE backed NHS use of AstraZeneca’s diabetes and heart failure drug Forxiga for the treatment of chronic kidney disease. pharmaphorum caught up with AZ’s Joris Silon to discuss unmet needs in the condition. “Many times, I have talked to nephrologists who take care of patients with chronic kidney disease in the later stages, […]

  • Biliary tract cancer drug granted orphan drug designation in US

    The US regulator has granted biliary tract cancer therapy Silmitasertib orphan drug designation. There are currently no treatments for this rare cancer, and the US food and Drug Administration (FDA) hopes that pharmaceutical firm Senhwa’s drug will meet the significant need for new biliary tract cancer options. First-line standard of care is currently a chemotherapy […]

  • Japan approves Pfizer COVID vaccine for children aged 5 to 11

    Japan’s health ministry has authorised the use of Pfizer and BioNTech COVID-19 vaccine in children aged between five and 11. The vaccine, Comirnaty, is claimed to be the first in Japan to be approved for use in this age group. Once the Ministry of Health, Labor and Welfare grants special approval, Japan will join the […]

  • UK green lights Gilead Sciences, Galapagos ulcerative colitis drug

    The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Jyseleca (filgotinib) 200mg tablets to treat ulcerative colitis (UC) in adults whose UC is moderately to severely active, with insufficient, lost response to, or who were not tolerant to either standard treatment or a biologic agent. Jyseleca, an oral preferential inhibitor of Janus Kinase […]

  • Glenmark and Lotus partner on allergic rhinitis spray 

    The mission to treat people who suffer from allergic rhinitis has been given a boost as two pharma companies join forces. The Swiss subsidiary of Glenmark Pharmaceuticals and Lotus International have signed a licensing agreement whereby Glenmark will handle the manufacture and supply of Ryaltris and Lotus will take charge of commercialising the drug, subject […]

  • Gilead takes legal action against counterfeit HIV drug network 

    US pharma giant Gilead Sciences has accused a network of unauthorised suppliers and distributors of selling more than $250 million of counterfeit versions of its HIV medications. According to the company, the defendants reportedly sold 85,247 bottles of medicine with counterfeit Gilead labelling to pharmacies over a two-year period, using falsified supply chain documentation to […]

  • Poolbeg licences candidate for broad-spectrum respiratory infections

    Poolbeg Pharma has licenced a candidate for nasal immunotherapy, developed by the University of Warwick, targeting respiratory infections, such as influenza and coronavirus. The RNA-based immunotherapy drug was developed at the University of Warwick and drew on 20 years of research, led by prominent virologists Professor Andrew Easton and Professor Nigel Dimmock. It will be developed by London-based Poolbeg as […]

  • Two new drugs added to WHO list of COVID-19 therapies

    A World Health Organization (WHO) panel has given the green light to two new drugs to treat COVID-19. US-based Eli Lilly’s baricitinib (Olumiant), a Janus kinase inhibitor, and GlaxoSmithKline and Vir Biotechnology’s sotrovimab, a monoclonal antibody, were recommended in new WHO guidelines, published in the British Medical Journal.  Baricitinib, administered with corticosteroids, was strongly recommended as […]

  • GSK rejects $68.4bn Unilever bid for consumer health

    GlaxoSmithKline (GSK) claims it has rejected three unsolicited, conditional and non-binding proposals from Unilever to acquire GSK Consumer Healthcare in a deal worth approximately $68.4 billion (£50 billion). The Consumer Healthcare business is a Joint Venture between GSK and Pfizer, with GSK holding a majority controlling interest of 68% and Pfizer 32%. The latest proposal, […]

  • Parallel Trade 2022

    SMi presents its 16th annual Parallel Trade conference on the 21st and the 22nd March 2022. As the only parallel trade conference in Europe, this event provides the perfect platform for industry experts from both sides of the coin to come together and share perspectives on the practice of parallel trade. The 2022 event will […]

  • Senate panel backs Biden’s FDA selection Robert Califf

    Robert Califf has cleared another stage on his return journey to the top job at the FDA, after the influential Senate committee on Health, Education, Labour and Pensions (HELP) voted by 13 to eight to support his nomination by President Joe Biden. Califf was nominated by Biden as FDA commissioner last November, ending a search […]

  • Third dose of AZ’s COVID jab boosts Omicron antibodies

    Preliminary data from an ongoing trial of AstraZeneca’s COVID-19 vaccine Vaxzevria has shown that a booster dose stimulates an increase in antibodies against Omicron, as well as other variants of the virus, including Alpha, Beta, Delta, and Gamma. The increased immune response was seen in people who received AZ’s shot as their primary vaccination as […]

  • Dream result for Idorsia as FDA clears its insomnia drug

    Swiss biotech Idorsia has claimed FDA approval for its first product – insomnia treatment Quviviq – setting up a market challenge to class rivals from Merck & Co and Eisai. The US regulator has cleared two doses of dual orexin receptor antagonist (DORA) Quviviq (daridorexant) – 25 mg and 50 mg – for the treatment […]

  • Nine for 2022: innovation and opportunities in healthcare

    In the second part of our examination of issues that will shape the course of 2022, we’ll update the ongoing impacts of the pandemic, which have, as they did in 2021, a pervasive effect on both pharma and healthcare issues, with themes we highlighted at the beginning of 2021 continuing their impact. Big Tech makes […]

  • Nine for 2022: International issues that are compelling, new or changed in 2022

    The year 2020 ended on a note of hope, with the 8th December vaccination of 91-year-old Margaret Keenan, the world’s first person to receive a COVID-19 vaccine outside clinical trials. Despite an estimated 9.25 billion doses administered globally by 4th January, with 25 different vaccines either approved for use or under emergency use authorisation, 2022 […]

  • After a Scottish aye, NICE says no to Lynparza for prostate cancer

    AstraZeneca’s PARP inhibitor Lynparza should not be made available routinely on the NHS n England and Wales as a treatment for prostate cancer, according to cost effectiveness agency NICE. That is the conclusion of draft guidance which says that the evidence for Lynparza (olaparib) as a treatment for prostate cancer is uncertain, and therefore not […]

  • Applied Therapeutics slumps as FDA seeks more data on lead drug

    Shares in Applied Therapeutics slumped today after it said it would have to delay a US filing for its lead drug programme AT-007, following an FDA request for more data. The New York-based biopharma company had been hoping to file AT007 in its lead indication galactosaemia in the third quarter of last year, but said […]

  • Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

    Biogen and Eisai head towards the end of the year with some much-needed good news in their Alzheimer’s programmes, as the FDA awards a fast-track designation to lecanemab, their follow-up to recently approved Aduhelm. The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of […]

  • The Considered pharmaceutical marketing experiment

    COVID-19 has changed everything – at least that’s David Hunt’s plan for pharmaceutical marketing. The former CEO of Havas Lynx recently launched a new agency in the US called The Considered, with the intent to break the traditional pharma marketing rules and shake up the industry. “There is some brilliant work that takes place by […]

  • Pfizer pips Merck to first oral COVID antiviral okay in US

    Pfizer’s Paxlovid has become the first oral antiviral for COVID-19 to be authorised for emergency use by the FDA, as the US braces itself for a surge in Omicron cases over the holiday period. The green light means that Paxlovid – based on nirmatrelvir (formerly PF-07321332) and ritonavir – has been cleared for use in […]

  • NICE backs Aimmune’s peanut allergy drug Palforzia for children

    Children in England with peanut allergies could be among the first in Europe to get access to Aimmune’s oral immunotherapy Palforzia, after the drugmaker agreed a supply deal with NHS England. The deal comes as cost-effectiveness watchdog NICE recommended Palforzia for NHS use in draft guidance published today which says the drug can be effective […]

  • Japan looks set to follow EU in rejecting Alzheimer’s drug Aduhelm

    The Japanese regulator looks like it could follow the European Commission and reject approval of Biogen and Eisai’s Alzheimer’s disease therapy Aduhelm, leaving the FDA an outlier in its stance on the drug. An advisory committee to the Ministry of Health, Labour and Welfare (MHLW) has declined to approve Aduhelm (aducanumab), saying the data from […]

  • 2021 in review: Building the framework of a patient-centric industry

    Patient centricity and patient engagement have been hot topics in pharma for some time – but buzz words do not always translate into concrete action. However, we have seen a framework of guidelines and recommendations during the last year on everything from increasing diversity to producing lay summaries, start to take shape. As part of […]

  • FDA knocks back Takeda’s blockbuster hope TAK-721

    Japanese drugmaker Takeda has suffered a blow to its late-stage pipeline, after the FDA rejected its marketing application for TAK-721, a drug candidate that is trying to be the first approved therapy in the US for eosinophilic oesophagitis (EoE). Takeda said it had received a complete response letter from the US regulator that is asking […]

  • FDA okays ViiV’s Apretude as first injectable for HIV PrEP

    GlaxoSmithKline’ majority-owned HIV unit ViiV has scored a key FDA approval, getting approval for its long-acting drug cabotegravir as the first injectable for pre-exposure prophylaxis (PrEP) –challenging market leader Gilead Sciences. Cabotegravir – given the trade name Apretude – can be dosed just six times a year and showed superior results to daily, oral therapy […]

  • Relief for AZ, Amgen as FDA okays severe asthma hope tezepelumab

    The FDA has approved AstraZeneca and Amgen’s severe asthma hope tezepelumab, exonerating the drugmakers’ decision to press ahead with a regulatory filing despite a failed phase 3 trial. Another phase 3 study supported the efficacy of the first-in-class TSLP inhibitor as an add-on treatment for people aged 12 and over with severe asthma, as did […]

  • Samantha Roberts to replace Gillian Leng as CEO of NICE

    Prof Gillian Leng’s replacement as chief executive of the National Institute for Health and Care Excellence (NICE) has been named as Dr Samantha Roberts, currently managing director for health and care at insurance group Legal and General. Roberts will take up the role on 1 February, as Leng retires from NICE after 20 years at […]

  • Biogen’s Alzheimer’s drug Aduhelm is rejected by EU advisors

    The EMA’s human medicines committee has said it cannot approve Biogen and Eisai’s Alzheimer’s disease therapy Aduhelm, saying it was unconvinced by the data submitted for the drug. The companies had already indicated that the CHMP looked likely to turn down their marketing application for Aduhelm (aducanumab), but the decision nevertheless is a heavy blow, […]

  • As Omicron cases rise, EMA backs early use of Pfizer’s Paxlovid

    The EMA’s human medicines committee has said that EU member states can start to offer Pfizer’s oral antiviral Paxlovid as a treatment for people with COVID-19 outside hospital, ahead of a formal approval for the drug. The decision comes amid rising rates of infection and deaths due to COVID-19 across the EU, and with 529 […]

  • NICE gives Keytruda parity with Opdivo for adjuvant melanoma

    After three years of availability via the Cancer Drugs Fund (CDF), MSD’s checkpoint inhibitor Keytruda has now been approved for routine NHS use after surgery for people with melanoma to prevent the cancer returning. PD-1 inhibitor Keytruda (pembrolizumab) has been recommended by NICE for adjuvant treatment of adults with stage 3 melanoma that has spread […]

  • EU gives narrower label to Apellis, Sobi’s PNH drug

    Apellis Pharma and partner Sobi have won EU approval for their paroxysmal nocturnal haemoglobinuria (PNH) drug pegcetacoplan in Europe, with a more restricted label than in the US. The European Commission has cleared the complement C3 inhibitor as Aspaveli for the treatment of adults with PNH who are anaemic, but only after they have been […]

  • Wales is first UK country to fund GW’s Epidyolex for tuberous sclerosis

    Patients in Wales with a rare disease that causes epilepsy will be the first in the UK to get access to GW Pharma’s cannabis-based medicine Epidyolex, after the drug was cleared for use by the NHS. Epidyolex (cannabidiol) can now be prescribed in Wales as a treatment for seizures associated with tuberous sclerosis complex (TSC), […]

  • FDA sets April date for verdict on Incyte’s vitiligo drug

    Incyte could be a few month away from a second FDA approval for its Opzelura cream that would make it the first medical treatment to re-pigment the skin of people with vitiligo. The FDA has kicked off a priority review of Opzelura (ruxolitinib), a JAK1/JAK2 inhibitor it approved to treat atopic dermatitis in September, and […]

  • Califf breezes through Senate nomination hearing

    Robert Califf looks set to take a second term as FDA commissioner after navigating a relatively placid two-hour confirmation hearing in the US Senate, with just a few ripples of discontent over issues like opioid medicines and his ties to the pharma industry. President Joe Biden’s nominated candidate for the post told lawmakers that he […]

  • Intercept’s travails continue as it pulls NASH drug filing in EU

    Already scrambling to refile its obeticholic acid (OCA) drug for non-alcoholic steatohepatitis (NASH) after the FDA rejected it last year, Intercept Pharma was hoping for more luck in Europe, but has now withdrawn its EU marketing application. Intercept had been hoping to extend the review period in the EU to allow time for results from […]

  • Post-Brexit medicines regulation must have international scope

    Post-pandemic, post-Brexit Britain must create a regulatory framework that both defines and aligns with international standards, or risk being left behind. If the UK creates regulatory frameworks that drastically diverge from those of its “science allies”, it could become a late – or even no – launch market for new treatments. The need for companies […]

  • AZ’s antibody first to be cleared by FDA for COVID prevention

    AstraZeneca’s antibody cocktail for COVID-19, now called Evushield, has become the first drug in the class to be authorised for prevention of infection by the US FDA. The long-acting antibody combination, formerly known as AZD7442 (tixagevimab and cilgavimab) has been cleared for pre-exposure prophylaxis (PrEP) of COVID-19 in adults and adolescents aged 12 and older […]

  • EU approves Roche’s Actemra for severe COVID-19

    The European Commission has moved swiftly to grant full approval to Roche’s Actemra/RoActemra as a treatment for severe COVID-19, clearing the drug within 24 hours of a recommendation from its advisory committee. Actemra (tocilizumab) has been approved for the treatment of COVID-19 in adults who are receiving systemic corticosteroids and require supplemental oxygen or mechanical […]

  • MHRA okays GSK’s Xevudy as data suggest it works against Omicron

    The UK medicines regulator has approved GlaxoSmithKline and Vir Biotech’s antibody for COVID-19, Xevudy, which has been shown to be effective against the new Omicron strain of SARS-CoV-2 in lab testing. Xevudy (sotrovimab) is the second COVID-19 antibody to be approved by the MHRA after Roche/Regeneron’s Ronapreve (casirivimab and imdevimab), which got a green light […]

  • Merck’s oral COVID drug squeaks past FDA advisory committee

    An FDA advisory committee voted by 13 to 10 to recommend emergency-use authorisation for Merck & Co and Ridgeback’s oral antiviral molnupiravir for mild-to-moderate COVID-19, saying it would be useful for some high-risk patients. The panel met shortly after updated results from the phase 3 MOVE-OUT trial of the drug showed it was less effective […]

  • FDA sets Sept review date for BMS’ psoriasis drug deucravacitinib

    The FDA has kicked off its review of Bristol-Myers Squibb’s psoriasis therapy deucravacitinib, setting an action date of 1 September next year, as the EU and Japanese regulators also start their appraisals of the drug. If approved, deucravacitinib would become the first tyrosine kinase 2 (TYK2) inhibitor approved for the treatment of any disease, said […]

  • Turkey’s home-grown COVID vaccine project nears fruition

    Turkey could have its own COVID-19 vaccine Turkovac available for use by the end of the year, according to the country’s Health Minister Fahrettin Koca. An emergency use authorisation (EUA) application for the inactivated SARS-CoV-2 vaccine has been submitted to the Turkish Medicines and Medical Devices Agency (TITCK) on the back of preliminary data from […]

  • After Scotland says no, NICE backs Lilly’s Retsevmo for lung cancer

    Eli Lilly’s RET inhibitor Retsevmo can now be prescribed on the NHS for people with RET fusion-positive advanced non-small cell lung cancer (NSCLC) in England and Wales, within the Cancer Drugs Fund (CDF). The recommendation from health technology assessment (HTA) agency NICE comes a couple of weeks after Retsevmo (selpercatinib) was cleared for use in […]

  • Innovation and collaboration – the MHRA’s pandemic approach

    The pandemic has propelled issues for the pharmaceutical industry to the top of the political and public agenda, from clinical trial recruitment to the journey required for medicines or vaccines to reach patients. The public knows now more than ever that the Medicines and Healthcare products Regulatory Agency (MHRA) ensures the safety and quality of […]

  • FDA clears Takeda drug for post-transplant CMV infections

    Takeda has scored the first-ever FDA approval of a treatment for refractory cytomegalovirus (CMV) infections in organ transplant patients, which can lead to an increased risk of complications and even death. Livtencity (maribavir) has been cleared for use in transplant patients aged 12 or over with CMV infections that do not respond to first-line treatment […]

  • Gilead files first hepatitis delta therapy with FDA

    Gilead Sciences has submitted its marketing application for bulevirtide, its antiviral therapy for rare hepatitis delta virus (HDV) infections, to the FDA. If approved, bulevirtide will be the first drug to be approved in the US for HDV, a virus that exists only as a co-infection with hepatitis B virus (HBV). It occurs in around […]

  • NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

    Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The cost-effectiveness agency had turned down Evrysdi (risdiplam) in draft guidance published in June, saying it was too expensive, but after negotiations with […]

  • NICE backs ViiV/J&J’s six-per-year injection for HIV

    People living with HIV in England and Wales now have an alternative to daily oral tablets, after NICE backed NHS use of two long-acting injectable therapies developed by ViiV Healthcare and Johnson & Johnson’s Janssen pharma unit. The cost-effectiveness watchdog has recommended the use of ViiV’s Vocabria (cabotegravir) with Janssen’s Rekambys (rilpivirine) as a combination […]

  • CHMP minded to turn down Alzheimer’s drug Aduhelm, says Biogen

    Biogen and Eisai have said that the EMA’s human medicines committee looks unlikely recommended approval of their Alzheimer’s drug Aduhelm when it comes up for discussion next month. The CHMP had a “negative trend vote” on the drug at a meeting last week which included an “oral explanation” of Aduhelm (aducanumab), which was approved in […]

  • NICE changes tack on NHS use of BMS’ myelofibrosis drug Inrebic

    After turning down routine NHS funding of Bristol-Myers Squibb’s Inrebic for bone marrow disorder myelofibrosis earlier this year, NICE now says the drug can be made available via the Cancer Drugs Fund. The health technology assessment (HTA) agency has recommended Inrebic (fedratinib) for treating disease-related splenomegaly (enlarged spleen) or other symptoms in myelofibrosis in patients […]

  • Pfizer files oral COVID pill in US, signs access deal for 95 countries

    Armed with impressive new data for its oral antiviral Paxlovid, Pfizer has filed for emergency use authorisation as a COVID-19 treatment in the US – and signed a deal to allow other manufacturers to make it in some lower-income countries. Paxlovid – based on new protease inhibitor PF-07321332 and HIV drug ritonavir – was found […]

  • Drug-resistant epilepsy drug Ontozry backed by NICE

    NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry. The cost-effectiveness agency has backed Ontozry (cenobamate) as an option for treating focal onset seizures with or without secondary generalised seizures in adults with drug-resistant […]

  • Biden confirms Califf choice as FDA commissioner

    President Joe Biden ended months of speculation by formally nominating Robert Califf as the new commissioner of the FDA, a position he previously held during the Obama administration. In a statement, Biden said Califf “has the experience and expertise to lead the FDA during a critical time in our nation’s fight to put an end […]

  • Novo Nordisk nears EU approval of obesity drug Wegovy

    Danish drugmaker Novo Nordisk is hoping to launch its once-weekly injectable treatment for obesity, Wegovy, in the EU next year after getting a green light from the EMA’s human medicines committee. The CHMP recommended marketing authorisation for Wegovy (semaglutide) for chronic weight management in adults with obesity, and also in overweight adult patients with a […]

  • COVID antibodies from Roche, Celltrion backed for approval in EU

    The EMA’s human medicines committee has recommended approval of Roche’s Ronapreve and Celltrion’s Regkirona for use in COVID-19, the first antibody-based therapies for coronavirus to be backed for full approval in the EU. Ronapreve (casirivimab and imdevimab) – developed and distributed by Regeneron as REGN-COV in the US – has been recommended by the CHMP […]

  • Getting better at measuring international uptake of medicines?

    In part one of a two-piece series, Leela Barham looks at whether we are getting better at measuring international uptake of medicines. The publication of a new report from healthcare analytics firm LOGEX, with the Institute for Rational Use of Medicine (IVM), on uptake in England should provide plenty of food for thought for the […]

  • J&J deal with COVAX grants humanitarian access to COVID shot

    Johnson & Johnson has reached an agreement to make its one-dose COVID-19 vaccine available to an arm of the COVAX facility that is focusing on providing shots to around 167 million displaced people around the world. World Health Organization-backed COVAX is already working hard to deliver vaccines to lower-income countries, but earlier this year set […]

  • Moderna squares off with US government over COVID jab patent

    After being hailed as a triumph of public-private drug development, Moderna’s partnership with the US government on its COVID-19 vaccine looks like it may descend into acrimony. That is according to a New York Times report, which says Moderna is now in dispute with the National Institutes of Health (NIH) after it filed a US […]

  • Germany says only Pfizer COVID jab should be used in under 30s

    Germany’s vaccination advisory committee has recommended that people aged under 30 should only be offered Pfizer/BioNTech’s COVID-19 vaccine Comirnaty, saying it seems less likely to cause heart inflammation than Moderna’s rival shot Spikevax. The draft guidance from the Robert Koch Institute’s STIKO committee also says that pregnant women, regardless of their age, should get the […]

  • Patient and market insights from leading KOLs and life science leaders

    Today, there are more than 80 treatments in development for atopic dermatitis and psoriasis. The landscape is booming with treatment innovations. However, the rise in research and development has created a crowded market that healthcare professionals struggle to navigate. Dermatologists face an abundance of data and more complex barriers to access than ever before. These […]

  • Cautious welcome as NICE backs Forxiga for chronic kidney disease

    Tens of thousands of people with chronic kidney disease in England could be eligible for treatment with AstraZeneca’s SGLT2 inhibitor Forxiga, after NICE backed NHS use of the drug in draft recommendations. AZ said that the decision is a “milestone” in the treatment of CKD, becoming the first new treatment option for patients in nearly […]

  • UK first to authorise Merck/Ridgeback’s COVID pill molnupiravir

    Merck & Co and Ridgeback’s oral antiviral molnupiravir has been approved in its first market – the UK – as a treatment for people with mild to moderate COVID-19 who are at increased risk of developing severe disease. The Medicines and Healthcare products Regulatory Agency (MHRA) said today that molnupiravir, now given the trade name […]

  • Novavax gets first authorisation for its COVID-19 jab

    Novavax has been granted its first emergency use authorisation in Indonesia for COVID-19 vaccine NVX-CoV2373, and expects other regulators in India, the Philippines and other countries to follow suit in the coming weeks. The vaccine is the first based on recombinant protein to be approved anywhere in the world, and has the key advantage over […]

  • FDA delays review of Moderna COVD jab in adolescents

    The FDA has said it needs more time to review Moderna’s application for emergency use of its COVID-19 vaccine mRNA-1273 in children aged 12 to 17, as it looks at data on heart inflammation in people receiving the shot. The decision leaves Moderna playing catch-up with Pfizer and BioNTech, whose Comirnaty vaccine was approved for […]

  • AbbVie claims first FDA okay for presbyopia drug therapy

    The FDA has approved the first and so far only pharmacological treatment for presbyopia, a sight condition that typically starts in middle age and involves difficulty in focusing on near objects. The approval has been granted to Vuity (formerly AGN-190584), a one-daily eyedrop formulation of muscarinic cholinergic agonist pilocarpine developed by AbbVie’s Allergen unit that […]

  • Merck takes further steps to expand access to oral COVID pill

    Merck & Co has signed a licensing deal with the UN-backed Medicines Patent Pool (MPP) that could pave the way for generic companies to manufacture its much-anticipated oral antiviral for COVID-19. The agreement would provide royalty-free licenses to companies manufacturing molnupiravir – originally developed by Ridgeback Biotherapeutics – for distribution in 105 low- and middle-income […]

  • Lilly files Alzheimer’s drug donanemab as Cortexyme rival fails trial

    Eli Lilly has followed through on its plan to file for approval of its Alzheimer’s disease candidate donanemab, and is planning to take on Biogen and Eisai with a head-to-head trial against their recently-approved Aduhelm drug. Lilly has also picked up an accelerated review for donanemab from the FDA, which is assessing the drug under […]

  • Seagen’s breast cancer drug Tukysa turned down by NICE

    NICE has decided not to recommend Seagen’s orally-active HER2 inhibitor Tukysa for advanced breast cancer in draft guidance, saying it isn’t a cost-effective use of NHS resources. Tukysa (tucatinib) was approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) in February as a third-line treatment for HER2-positive breast cancer, shortly after it was […]

  • Sustained healthcare systems need sustainable pharma business models

    COVID-19 demonstrated the importance of robust supply chains and the dangers of an over reliance on overseas manufacturing. But can the Pharmaceutical Strategy for Europe help to redress the balance? Only sustainable, robust supply chains and business models will allow innovation to flourish, secure access to medicines, and minimise the industry’s impact on the environment. […]

  • EU starts review of Merck’s oral COVID drug molnupiravir

    The EMA has started a rolling review of Merck & Co and Ridgeback Biotherapeutics’ much-anticipated oral antiviral molnupiravir as a treatment for COVID-19 in adults. Molnupiravir is already filed for approval in the US and Canada – where Merck is known as MSD – amid hopes that the drug could be used to protect vulnerable […]

  • FDA clears Roche’s Susvimo implant for eye disease wet AMD

    Roche’s Genentech unit has won FDA approval for Susvimo, an implant for eyesight-robbing disease wet age-related macular degeneration (AMD) that gives patients an alternative to regular injections into the eye. Susvimo has the same active ingredient as Roche’s big-selling therapy Lucentis (ranibizumab), a VEGF inhibitor administered once a month that has been approved by the […]

  • Alnylam gets NICE backing for porphyria therapy Givlaari

    UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. Givlaari (givosiran) has been recommended as a treatment option for people aged 12 and over with AHP, a […]

  • Biogen, Sage prep 2022 filing for depression drug zuranolone

    The renaissance of Biogen and Sage Therapeutics antidepressant zuranolone continues, with the companies indicating they now plan to start a rolling submission for the drug early next year. Two years ago things were looking bleak for zuranolone after the oral GABAA receptor modulator failed the phase 3 MOUNTAIN trial in major depressive disorder (MDD), raising […]

  • Omeros seeks urgent FDA meeting after it rejects key drug

    Shares in Omeros plummeted yesterday after the FDA rejected approval of its narsoplimab drug for a rare and life-threatening complication of haematopoietic stem cell transplants (HSCT), saying it was struggling to gauge the treatment effect of the drug. The biotech is seeking a so-called ‘Type A’ meeting with the FDA to see how the application […]

  • NICE changes stance on Sanofi’s Dupixent for severe asthma

    18 months after saying “no”  to regular NHS funding for Sanofi and Regeneron’s Dupixent (dupilumab) for severe asthma in initial guidance, NICE has now backed the drug. The final appraisal document for Dupixent (dupilumab) has given the green light to use of the drug as an add-on maintenance therapy for people aged over 12 with […]

  • COVID-19 has propelled the regulatory industry years ahead

    Janssen’s Saskia De Haes, VP EMEA Regulatory Affairs, looks at how the pandemic response has shaped tomorrow’s regulatory landscape. In my 26 years of industry experience, I’ve often found the regulatory process to have  plenty of opportunities to become more efficient and faster. When the COVID-19 pandemic hit, it lit a fire under the regulatory […]

  • Califf re-joining FDA would be positive for industry; analyst

    With rumours growing that that ex-FDA head Robert Califf looks set to receive President Joe Biden’s nomination to be the next FDA commissioner, at least one analyst thinks the appointment would be a positive for the biopharma industry. Califf was appointed to the helm of the FDA under the Obama administration in 2016, and picked […]

  • Merck files pill for milder COVID, as AZ showcases its antibody data

    As expected, Merck & Co has moved ahead swiftly with an emergency use application for its oral antiviral molnupiravir, aiming to protect people with mild or moderate COVID-19 from developing severe disease with a drug that can be delivered outside hospital. Merck & partner Ridgeback Therapeutics have filed for approval to use molnupiravir in adults […]

  • Scotland backs Cabometyx combo for kidney cancer, amid stalled NICE review

    Patients in Scotland with newly-diagnosed advanced kidney cancer can now access a therapy combining Bristol-Myers Squibb’s immunotherapy Opdivo with Ipsen’s targeted drug Cabometyx, ahead of the rest of the UK and most other countries in the world. The Scottish Medicines Consortium (SMC) has backed Opdivo (nivolumab) plus Cabometyx (cabozantinib) as a first-line therapy for people […]

  • Flash FDA approval sends ChemoCentryx spiralling upwards

    ChemoCentryx has claimed FDA approval for its avacopan drug for a rare autoimmune disease, becoming the first orally-active drug that targets the complement C5 receptor. Avacopan will be launched as Tavneos in the coming weeks as a treatment for two forms of ANCA vasculitis – granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) – in […]

  • FDA panel backs Takeda’s CMV drug for transplant patients

    Takeda is closing on a first FDA approval in the treatment of refractory cytomegalovirus (CMV) infections in organ transplant patients, after agency advisors voted unanimously in favour of its antiviral maribavir. Maribavir (TAK-620) should be approved for refractory CMV infections in people with solid organ transplants as well as haematopoietic stem cell transplants (HSCT), regardless […]

  • Consternation as NICE rejects Janssen MS drug Ponvory

    NICE has delivered its first verdict on NHS funding of Janssen’s oral multiple sclerosis drug Ponvory, and it’s not good news for patients with the disease waiting for new treatment options. In a provisional decision, NICE has said Ponvory (ponesimod) should not be available routinely on the NHS in England and Wales for relapsing forms […]

  • Moderna unveils $500m plan for African vaccine facility

    Moderna has said it plans to invest $500 million in a new facility for manufacturing mRNA vaccines in Africa, and has kicked off a search to find a suitable country and location. The factory will be able to supply up to 500 million doses of mRNA shots a year, and cover the full spectrum of […]

  • J&J joins COVID-19 booster shot drive, filing with FDA

    Johnson & Johnson’s COVID-19 vaccine has been a minor player in the US vaccination drive so far, but the company hopes it can have a bigger role as a booster shot. The drugmaker has filed for approval of a second dose of its one-shot Ad26.COV2.S vaccine for people aged 18 years and older, based on […]

  • AZ files for emergency use of COVID-19 antibody combo in US

    AstraZeneca has sought emergency use authorisation for its antibody combination AZD7442, following trial results indicating it can prevent symptomatic COVID-19 infection when given to healthy patients. AZD7442 (tixagevimab/cilgavimab) is the first long-acting antibody drug that has demonstrated it can be used in this way, reducing the risk of symptomatic COVID-19 by 77% compared to placebo […]

  • Joy as sickle cell patients get access to Novartis’ Adakveo after NICE ruling

    People living with sickle cell disease (SCD) in England and Wales will be able to get access to treatment with Novartis’ Adakveo, thanks to a special arrangement between the drugmaker and NICE. The decision makes Adakveo (crizanlizumab) the first new treatment for SCD available via the NHS for 20 years, according to the cost-effectiveness agency, […]

  • NICE clears NHS use of Roche’s Tecentriq in bladder cancer

    NICE has recommended routine NHS access to Roche’s cancer immunotherapy Tecentriq for some bladder cancer patients, four years after it was first made available via the Cancer Drugs Fund (CDF). The PD-L1 inhibitor was initially turned down by NICE for a form of bladder cancer called urothelial carcinoma (UC), but Roche was encouraged to make […]

  • AbbVie’s Qulipta cleared for migraine prevention, setting up Biohaven clash

    AbbVie has added a third drug to its arsenal in the crowded migraine market, getting FDA approval for Qulipta – its once-daily oral preventive therapy for episodic migraines. Qulipta (atogepant) is AbbVie’s second oral CGRP inhibitor after Ubrelvy (ubrogepant) – which was approved for the acute treatment of migraine attacks in 2019 – and rounds […]

  • Biogen, Eisai double down on Alzheimer’s with lecanemab filing

    Biogen and Eisai’s first Alzheimer’s disease drug Aduhelm is facing an uphill battle in the US market, but the partners are forging ahead with their second candidate lecanemab, starting a rolling submission to the FDA. Like Aduhelm (aducanumab) lecanemab – also known as BAN2401 – is another amyloid-directed antibody but has a slightly different mechanism, […]

  • Takeda bags Japanese approval for Crohn’s cell therapy Alofisel

    Takeda has chalked up another milestone for its Alofisel cell therapy for a complication of Crohn’s disease, becoming the first allogeneic stem cell therapy to be approved in Japan. Alofisel (darvadstrocel) has been cleared by the Ministry of Health, Labour and Welfare (MHLW) for the treatment of complex perianal fistulas in patients with non-active or […]

  • CDC overrules its advisors, backing COVID booster for health workers

    The Centre for Disease Control and Prevention (CDC) in the US has backed booster doses of Pfizer and BioNTech’s COVID-19 vaccine Comirnaty in line with its recent FDA approval, overruling the advice of its own expert advisors. The CDC’s Advisory Committee on Immunisation Practices (ACIP) voted unanimously yesterday to recommend a third dose of Comirnaty […]

  • Novavax files its COVID jab to WHO for emergency use listing

    Novavax and its partner Serum Institute of India have applied to the World Health Organization for an emergency use listing for their COVID-19 vaccine, based on a conventional recombinant protein technology. Getting a WHO emergency use listing (EUL) is a requirement for approvals by many national regulatory authorities, as well as for participation in the […]

  • FDA approves Pfizer COVID booster jabs for higher-risk groups

    Just days after its advisory committee meeting voted to approve a third dose of Pfizer and BioNTech’s COVID-19 vaccine, the FDA has followed through and cleared the shot. The FDA has amended the emergency use authorisation (EUA) for Comirnaty in line with its experts’ advice – for people aged over 65 and other adults at […]

  • Incyte leads JAK push into eczema as FDA clears Opzelura cream

    The FDA has approved Incyte’s Opzelura cream for atopic dermatitis, commonly called eczema, becoming the first and only topical JAK inhibitor registered in the US. Opzelura (ruxolitinib) – which has been cleared to treat atopic dermatitis in adolescents and adults whose symptoms cannot be treated using other topical therapies – has beaten its orally-administered rivals […]

  • FDA OKs first biosimilar of Roche’s blockbuster AMD drug Lucentis

    Samsung Bioepis and Biogen have claimed the first FDA approval for a biosimilar version of Roche and Novartis’ Lucentis (ranibizumab) for leading causes of blindness, raising the prospect of a cheaper treatment option for US patients. The US green light for Byooviz (formerly SB11) covers the treatment of wet age-related macular degeneration (AMD) and two […]

  • EU panel rejects Pfizer’s tanezumab for osteoarthritis pain

    Any lingering hope that Pfizer’s may finally get a regulatory approval for its nerve growth factor (NGF) inhibitor tanezumab looks to have been dashed after the EMA’s human medicines committee rejected the drug. A positive recommendation was already looking like a long shot for after FDA advisors voted 15 to one against approval of the […]

  • ICER says Amgen’s tezepelumab ‘likely to be too expensive’

    Amgen and AstraZeneca haven’t won approval or announced a price for their severe asthma therapy tezepelumab should it reach the market, but ICER in the US maintains it will probably not be cost-effective. The cost effectiveness organisation has published a draft evidence report on the first-in-class TSLP inhibitor – which is currently under FDA review […]

  • Keytruda backed by NICE for late-stage oesophageal cancer

    MSD’s Keytruda has become the first immunotherapy cleared by NICE for previously-untreated patients with advanced oesophageal cancer, making it an option for routine NHS treatment. Keytruda (pembrolizumab) has been given the green light for use alongside platinum- and fluoropyrimidine-based chemotherapy in patients with locally advanced or metastatic oesophageal cancer that cannot be treated with surgery. […]

  • Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval

    Takeda has had a run of bad luck with its Wave1 pipeline of new drug candidates of late, but can now celebrate a vestry after getting FDA approval for first-in-class lung cancer therapy Exkivity. The US regulator has cleared the tyrosine kinase inhibitor (TKI) as a second-line treatment for locally advanced or metastatic non-small-cell lung […]

  • NICE backs BioCryst’s Orladeyo for hereditary angioedema

    BioCryst Pharma’s Orladeyo has been recommended by NICE as a treatment for the rare disease hereditary angioedema (HAE) in England, Wales and Northern Ireland. Orladeyo (berotralstat) – which was approved by the EMA and MHRA in April and May, respectively – is set to become the first oral, once-daily therapy that can be used to […]

  • Ahead of FDA adcomm, Pfizer, Moderna say COVID jab efficacy dips

    Pfizer and Moderna have both reported data that they say backs up the need for booster shots of their COVID-19 vaccines ahead of an FDA advisory committee meeting tomorrow, although the regulator seems less convinced. Documents published ahead of the panel by the FDA reviewers suggest that there is some evidence from observational studies that […]

  • Amylyx preps filing for its ALS drug after FDA feedback

    Just a few months ago, Amylyx was talking about having to complete another clinical trial before it could file for FDA approval of its drug for neurodegenerative disease amyotrophic lateral sclerosis (ALS). It’s now accelerating those plans, citing favourable discussions with the US regulator. The company revealed today that it will submit its marketing application […]

  • ObsEva files uterine fibroid drug in US, chasing AbbVie, Myovant

    Swiss biopharma company ObsEva has filed its oral GnRH antagonist linzagolix for the treatment of uterine fibroids in the US, hoping to expand the number of women eligible for this type of therapy. If approved, linzagolix will be the only drug in the class with a dosing regimen intended for women with uterine fibroids who […]

  • Pfizer gets first approval, in UK, for Xeljanz follow-up Cibinqo

    Pfizer has claimed its first regulatory approval worldwide for Cibinqo, its JAK inhibitor for atopic dermatitis, from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). The green light covers England, Scotland Wales, as Northern Ireland remains under the EU regulatory framework, and covers once-daily use of the orally-active drug in adolescents aged over 12 […]

  • MHRA cuts could affect UK regulatory decisions, say unions

    A 20% to 25% reduction in staffing at the UK Medicines and Healthcare products Regulatory Agency (MHRA) risks undermining the ability of the authority to fulfil its role, according to trade unions. In an open letter to Health Secretary Sajid Javid, Prospect, Unite and three other unions say the decision to slash the MHRA’s current […]

  • NICE changes its mind on J&J’s Erleada in prostate cancer

    NICE has reversed its position on Johnson & Johnson’s oral prostate cancer therapy Erleada in patients with hormone-relapsed or hormone sensitive tumours, backing NHS use of the drug. The change of heart comes after J&J’s Janssen pharma unit offered an improved discount on Erleada (apalutamide), according to the cost-effectiveness watchdog in its final appraisal determination, […]

  • EMA looks at booster data for Pfizer/BioNTech COVID-19 jab

    The EU regulator has started to review a marketing application filed by Pfizer and BioNTech for a third, booster dose of their Comirnaty COVID-19 vaccine given six months after the first course to people aged 16 and over. The EMA’s CHMP human medicines committee will carry out an accelerated review of the application, which comes […]

  • NICE backs Cosentyx’ use in thousands of children with psoriasis

    NICE has recommended broadening NHS use of Novartis’ IL-17 inhibitor Cosentyx to include children aged six to 17 with severe plaque psoriasis. The cost-effectiveness agency says in draft guidance that Cosentyx (secukinumab) should be offered as an option to patients in this age bracket who either don’t respond to or cannot tolerate other systemic treatments. […]

  • FDA schedules adcomm for Pfizer’s COVID booster filing

    The FDA has said it will hold an advisory committee meeting later this month to discuss Pfizer and BioNTech’s filing for approval of a third booster dose of their COVID-19 vaccine Comirnaty in people aged 16 and over. The adcomm is scheduled for 17 September and will discuss the Pfizer/BioNTech filing as well as booster […]

  • NICE backs NHS use of Novartis’ cholesterol drug Leqvio

    Novartis’ cholesterol-lowering drug Leqvio will be made available on the NHS in England and Wales, and could help to prevent up to 30,000 deaths, according to NICE. The PCSK9-targeting oligonucleotide – which is already available in Scotland – requires dosing just twice a year and has been approved by the health technology assessment (HTA) agency […]

  • NICE rejects J&J’ Darzalex regimen for multiple myeloma

    New guidance from NICE has rejected a combination regimen based on Johnson & Johnson’s Darzalex for a group of previously-untreated patients with multiple myeloma, a type of bone marrow cancer. The draft document covers the use of Darzalex (daratumumab) as an add-on to standard induction treatment with Takeda’s Velcade (bortezomib) plus thalidomide and dexamethasone given […]

  • AbbVie’s Rinvoq is first JAK drug cleared in EU for atopic dermatitis

    The FDA may be reluctant to trust JAK inhibitors, but the European Commission is more willing, and has just handed a first-in-class approval in atopic dermatitis to AbbVie’s Rinvoq. The EU regulator has cleared Rinvoq (upadacitinib) as an oral treatment for moderate to severe atopic dermatitis in adults and adolescents 12 years and older, either […]

  • Cara’s Korsuva is first drug cleared for itching in dialysis patients

    One of the most common and challenging side effects for people undergoing dialysis for chronic kidney disease – chronic itching – now has an FDA-approved treatment. The US regulator has cleared Cara Therapeutics’ Korsuva (difelikefalin) as an injectable treatment for chronic itching, also known as pruritus, in these patients after a priority review. The green […]

  • IL-17 tailender UCB gets EU OK for bimekizumab in psoriasis

    UCB has been bringing up the rear in the IL-17 inhibitor category with bimekizumab, but now has EU approval for the drug and thinks it can mount a stiff challenge to the leaders, including Novartis’ big-selling Cosentyx. The European Commission has cleared the drug as Bimzelx for the treatment of moderate to severe plaque psoriasis […]

  • FDA grants full approval to Pfizer/BioNTech COVID jab

    Pfizer and BioNTech’s COVID-19 vaccine was the first to get emergency use authorisation (EUA) from the FDA, and has also become the first to get full regulatory approval in the US. The regulator has cleared the vaccine – now officially given the trade name Comirnaty – to prevent COVID-19 in people aged 16 or more, […]

  • COVID prompts another milestone, as India clears first DNA vaccine

    The COVID-19 pandemic accelerated the development of mRNA-based vaccines, and its influence has now extended to DNA-based shots as well, with Zydus Cadila’s ZyCoV-D getting emergency use authorisation in India. This is the first DNA vaccine to ever receive regulatory authorisation anywhere in the world, the first COVID-19 jab approved in India to treat children […]

  • After FDA snub, EMA backs FibroGen, Astellas’ roxadustat

    The EU regulator has approved  FibroGen and Astellas Evrenzo for adults with anaemia caused by chronic kidney disease (CKD), just days after the FDA turned down the drug in the US. The decision makes Evrenzo (roxadustat) the first oral HIF-PH inhibitor to be cleared for use in the EU, ahead of rival candidates daprodustat from […]

  • NICE says yes to Novartis’ Rydapt for rare blood disorder

    Novartis’ Rydapt has become the first and only licensed treatment for rare and life-threatening blood disorder systemic mastocytosis (SM) to be cleared for routine NHS use, after getting a green light from NICE. The decision means around 170 people with advanced SM in England and Wales will be eligible for  treatment with oral, twice-daily drug, […]

  • Jardiance gets parity with Farxiga in US as FDA clears use in heart failure

    Eli Lilly and Boehringer Ingelheim have claimed FDA approval for their diabetes therapy Jardiance in heart failure with reduced ejection fraction (HFreF), setting up a market tussle with AstraZeneca’s Farxiga. The US regulator has cleared a 10mg daily dose of Jardiance (empagliflozin) to reduce the risk of cardiovascular death plus hospitalisation for heart failure in […]

  • Amid Actemra shortage, WHO asks Roche to ensure equitable access

    Roche’s arthritis drug Actemra has become a key treatment for people with severe COVID-19, leading to shortages, and the World Health Organisation (WHO) wants the company to ensure that supplies don’t just go to wealthy countries. Earlier this week Roche’s Genentech unit said it was facing unprecedented demand for Actemra (tocilizumab) – known as RoActemra […]

  • Merck kicks off oral COVID-19 antiviral filing in Canada

    Health Canada has started a rolling review of Merck & Co’s molnupiravir, an oral antiviral therapy treatment for COVID-19 partnered with Ridgeback Biotherapeutics. The Canadian regulator will start reviewing early safety, quality and efficacy data for the drug while late-stage clinical trials are still underway, in the hope of approving the drug quickly of results […]

  • Ipsen hit by new palaver for rare disease drug palovarotene

    Just as Ipsen’s $1 billion bet on rare bone disease drug palovarotene seemed to be nearing the finish line, it has fallen at the final hurdle. After a string of setbacks with the programme, Ipsen finally filed palovarotene with the FDA in May, earning a priority review. But the French drugmaker has already withdrawn it, […]

  • Health insurer Excellus will not fund Alzheimer’s drug Aduhelm

    Health insurer Excellus BlueCross BlueShield has said it will not cover treatment with Biogen and Eisai’s new Alzheimer’s disease drug Aduhelm because it has not been shown to be medically effective. The company – said to be the largest health insurer in New York – said in a statement that Aduhelm (aducanumab) remains an investigational […]

  • A problem combination?

    Leela Barham takes stock of past NICE decisions for combinations to explore the scale of the reimbursement challenge for combination therapies. Combination therapies are now common in therapy areas like cancer. There have been cases where even if a new treatment used in combination were offered at zero cost to the NHS that it would […]

  • NICE changes stance on Lilly’s breast cancer dug Verzenio

    After rejecting Eli Lilly’s Verzenio for breast cancer therapy earlier this year, NICE has relented, recommending the drug for routine NHS use alongside hormonal therapy fulvestrant. A final appraisal document from the health technology assessment (HTA) agency backs the use of Verzenio (abemaciclib) alongside fulvestrant for the treatment of women with hormone receptor-positive, HER2-negative breast […]

  • FDA rejects FibroGen/AZ’s roxadustat, asking for new trial

    The FDA has demanded another clinical trial of FibroGen and AstraZeneca’s roxadustat for anaemia caused by chronic kidney disease (CKD) before it will consider approval, setting up a lengthy delay to the programme. The need for a new trial is something of a worst-case scenario for roxadustat, although a rejection was expected after an FDA […]

  • Axsome faces delay for depression drug, hitting shares hard

    The FDA was supposed to be delivering its verdict on Axsome Therapeutics’ depression therapy AXS-05 in less than a fortnight – but instead has delivered the company a letter outlining deficiencies in its marketing application. The problem for the company is that it doesn’t yet know what those deficiencies are, and that uncertainty weighed heavily […]

  • Collaborate to simplify cancer trials and accelerate post-pandemic recovery

    How could simplifying the administration of clinical trials help cancer diagnosis, care, and treatment levels to exceed pre-pandemic levels? Amanda Barrell reports from the ABPI Scotland cancer roundtable discussion. Work to get Scotland’s cancer services back to full strength following the pandemic has already begun, and the pharmaceutical industry has pledged to play its part. […]

  • Scotland backs Bavencio for bladder cancer after NICE says no

    Patients with bladder cancer in Scotland will be the first in the UK to claim access to maintenance treatment with Merck Serono and Pfizer’s Bavencio, ahead of their counterparts in England and Wales. The Scottish Medicines Consortium (SMC) has cleared NHS funding for Bavencio (avelumab) as a first-line maintenance therapy for people with advanced urothelial […]

  • Sanofi extends its Pompe range with first Nexviazyme OK

    Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved Nexviazyme (avalglucosidase alfa) for the treatment of patients aged one year of age and older with late-onset Pompe disease, which progressively attacks the heart and […]

  • Lilly sets out its stall on donanemab as filing approaches

    The FDA’s approval of Biogen’s Aduhelm for Alzheimer’s disease emboldened Eli Lilly to move forward plans to file its own candidate donanemab later this year, although it has acknowledged it will have to overcome reservations about anti-amyloid drugs. Lilly’s chief scientific officer Dan Skovronsky said on the company’s second-quarter results call that the results of […]

  • UCB’s bimekizumab ‘pre-approved’ using NICE fast-track scheme

    UCB’s bimekizumab hasn’t been approved for marketing in Europe yet, but has already been given the nod for NHS use in England and Wales under a new fast-track scheme introduced by NICE. The IL-17 inhibitor is the first to go through a new process aimed at accelerating appraisals for drugs deemed to be low-risk, which […]

  • FDA approves AZ’s anifromulab, ending 10-year lupus drug drought

    AstraZeneca has completed the resurrection of its anifrolumab drug for systemic lupus erythematosus (SLE) after claiming FDA approval for the drug as an add-on therapy for adults with the autoimmune disease. Anifrolumab was all-but written off in 2018 after failing the phase 3 TULIP-1 trial, but bounced back with positive data from a second study […]

  • FDA starts review of Roche’s eye disease drug, setting up 2022 verdict

    The FDA has started a priority review of Roche’s bispecific antibody faricimab for two major causes of blindness, preparing for a market showdown with Bayer and Regeneron’s market-leading Eylea. The US regulator will review the drug for both neovascular or ‘wet’ age-related macular degeneration (AMD) and diabetic macular oedema (DME), as well as diabetic retinopathy […]

  • FDA okays Mylan insulin drug as first ‘interchangeable’ biosimilar

    Generic drugmaker Mylan has become the first company to secure FDA approval for a biosimilar product that is considered completely interchangeable with the reference product – namely Sanofi’s once-daily insulin Lantus. Mylan’s Semglee has been approved for over a year as a regular biosimilar to Lantus (insulin glargine), meaning that it could be used in […]

  • Boost for AZ as Ultomiris gets CHMP nod for expanded label

    Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion of the label of Ultomiris, one of the main assets behind the $39 billion merger. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has backed the approval of Ultomiris (ravulizumab) for children and adolescents with […]

  • NHS sets aside £340m for new Innovative Medicines Fund

    NHS England has matched its financial backing of the Cancer Drugs Fund (CDF) with an equivalent pot of money for a new Innovative Medicines Fund (IMF) that will provide early access to cutting-edge medicines like cell and gene therapies. It has proposed that the IMF will work in the same way as the CDF, funding […]

  • Roche ‘talking to FDA’ as Alzheimer’s drug moves towards 2022 readout

    Phase 3 results with Roche’s amyloid-targeting Alzheimer’s drug gantenerumab may not be due until the second half of last year, but the company is already in discussions with the FDA about a route to approval. There has been speculation since the unexpected approval of Biogen and Eisai’s Aduhelm (aducanumab) last month that other amyloid therapies […]

  • NICE changes its mind on rare bile duct cancer drug from Incyte

    Incyte’s Pemazyre has become the first targeted therapy available for NHS patients with cholangiocarcinoma or bile duct cancer, after NICE concluded it provided value for money. The green light for Pemazyre (pemigatinib) has been hailed by cholangiocarcinoma charity AMMF, which said it was a “major milestone” in the fight against the “Cinderella” cancer, which for […]

  • Bluebird claims EU nod for rare disease therapy Skysona

    Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The EMA has cleared Skysona (elivaldogene autotemcel) for use in CALD patients aged under 18 who have an ABCD1 genetic mutation and no matched […]

  • Novartis eyes New Year verdict from FDA on delayed Leqvio

    Novartis has revealed that the FDA has accepted its resubmission for cholesterol-lowering drug Leqvio – rejected unexpectedly by the US regulator last year – and has set a new action date of 1 January 2022. The drugmaker refiled its application for Leqvio (inclisiran) earlier this month, trying to get the programme back on track in […]

  • EMA starts review of latecomer GSK/Sanofi COVID-19 vaccine

    The EMA has kicked off a rolling review of a COVID-19 vaccine from Sanofi and GlaxoSmithKline, which started a 35,000-patient phase 3 trial in May. The vaccine – now dubbed Vidprevtyn – is based on a recombinant protein antigen developed by Sanofi’s vaccines unit Sanofi Pasteur, and also includes an immune-boosting adjuvant developed by GSK […]

  • Hard times at Ardelyx as FDA rejects kidney disease drug tenapanor

    The FDA has taken a longer than usual look at Ardelyx’ regulatory submission for tenapanor, a drug for high blood phosphate levels associated with chronic kidney disease (CKD), and found it wanting – sending the biotech’s shares into a tailspin. The US regulator had extended its review of the drug by three months – setting […]

  • FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy

    Bayer subsidiary BlueRock Therapeutics has been granted a fast-track review by the FDA for DA01, its stem cell-based therapy for Parkinson’s disease which is currently in early-stage clinical testing. The FDA designation allows for benefits such as more frequent meetings and communication with the regulator during clinical development, and a truncated six-month review time. Those […]

  • FDA okays Merck’s would-be Prevnar rival Vaxneuvance

    The FDA has approved a new pneumococcal vaccine from Merck & Co, just over a month after Pfizer got a green light for a new version of its market leading Prevnar shot. Merck’s Vaxneuvance – formerly known as V114 – is the first major competition to Pfizer’s franchise in the big market for conjugate pneumococcal […]

  • FDA adcomm turns down FibroGen, AZ’s roxadustat for CKD anaemia

    Safety concerns seem to have scuppered any hope of a near-term approval for FibroGen and AstraZeneca’s roxadustat for anaemia associated with chronic kidney disease (CKD) in the US, after FDA advisors voted comprehensively against the drug yesterday. Just one of the 14-member panel thought that roxadustat should be approved for non-dialysis-dependent CKD patients, and only […]

  • Merck’s targeted lung cancer drug tepotinib wins early access in UK

    The UK drugs regulator has backed Merck KGaA’s MET inhibitor for the treatment of some patients with non-small cell lung cancer under the early access to medicines scheme (EAMS). The EAMS – first introduced in 2014 – provides a route for drugmakers to provide earlier availability of promising new unlicensed medicines to patients with few […]

  • NICE feedback?

    The National Institute for Health and Care Excellence (NICE) is quite far along a NICE-led process to review its methods and processes for evaluating technologies. As part of that work, NICE is engaging widely with stakeholders, including patient organisations. Leela Barham provides an independent view of patient organisations responses to a NICE online survey and […]

  • US starts review of Aduhelm coverage amid fears of Medicare hit

    The US government has started a review of how Biogen and Eisai’s pricey new Alzheimer’s disease therapy Aduhelm will be covered by Medicare, to see if a national strategy needs to be adopted. At the moment, the coverage for $56,000-a-year Aduhelm (aducanumab) is currently determined at the local level by Medicare administrative contractors representing 12 […]

  • Bayer’s finerenone gets FDA nod for diabetic kidney disease

    Bayer’s big investment in time and money on the development of finerenone for chronic kidney disease (CKD) in diabetics has paid off with an FDA approval, although it could face stiff competition in the market.  The oral, non-steroidal mineralocorticoid receptor (MR) antagonist has been approved as Kerendia in the US – its first world market […]

  • Aduhelm approval controversy dials up as FDA seeks probe

    In another twist to the Aduhelm approval tale, FDA’s Janet Woodcock has called for an independent investigation into the relationship between agency staffers and Biogen executives. The acting FDA commissioner has sent a letter to the Department of Health and Human Services (HHS) inspector general Christi Grimm on Friday, asking for a review of the […]

  • How digital pathways are changing healthcare

    As demonstrated throughout COVID, digital health is no longer experimental. Digital technologies are proving their value by allowing for better care, improved adherence and a more connected ecosystem between doctors, patients and pharma. Ampersand Health has been working in the space since 2015 and has seen it evolve from a niche approach with limited acceptance […]

  • NICE rejects Orchard’s gene therapy for rare childhood disease MLD

    Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there was evidence of a short-term benefit with Libmeldy (atidarsagene autotemcel or OTL-200), assumptions about its long-term effects are uncertain, making it unclear whether it will offer […]

  • Eyeing delta surge, Pfizer will file COVID-19 booster next month

    With the COVID-19 delta variant has become the dominant strain circulating in the US, Pfizer and BioNTech have said they will ask the FDA and other regulators to approve a third dose of their Comirnaty vaccine to guard against a winter surge in cases.  The two companies have revealed data from an ongoing trial of […]

  • FDA whittles back Aduhelm approval amid concern over broad label

    One of the criticisms levelled at the FDA over its approval of Biogen and Eisai’s Alzheimer’s disease drug Aduhelm was its decision to clear use of the drug in a broader group of patients than was included in clinical trials. Now, it has backtracked on that decision. The updated label for Aduhelm (aducanumab) says it […]

  • AZ, Amgen get speedy FDA review for asthma drug tezepelumab

    The FDA has started a priority review of AstraZeneca and Amgen’s severe asthma antibody tezepelumab, setting a date for a decision in the first quarter of 2022. If approved, TSLP inhibitor tezepelumab could reach the market with a broader label than currently-used asthma antibodies, which are approved to treat patient with severe asthma characterised by […]

  • A life sciences vision to deliver health, wealth and resilience for the UK

    The ABPI’s chief executive Dr Richard Torbett outlines a new ‘vision’ and 10-year strategy for UK life sciences, launched by the government today. After an extraordinary year of science versus disease, the UK has cemented itself at the forefront of the global response to COVID-19. What has been achieved in just 18 months is nothing […]

  • FDA rejects Provention’s type 1 diabetes drug teplizumab

    The FDA indicated something was not well with Provention Bio’s filing for type 1 diabetes therapy teplizumab in April when it delayed its review. Now, the agency has rejected the application entirely, sending the company’s shares into a steep slide. The complete response letter (CRL) for teplizumab as a treatment to delay clinical type 1 […]

  • Novartis tries to rescue stalled inclisiran filing with FDA

    Novartis has tried to get its marketing application for high cholesterol therapy inclisiran in the US back on track, after the FDA rejected it last year, by changing the factory that makes the drug. The US regulator issued Novartis with a complete response letter (CRL) for inclisiran in December, citing “unresolved facility inspection-related conditions” at […]

  • Zydus Cadila nears world-first human plasmid DNA vaccine approval

    Zydus Cadila has filed for emergency use approval (EUA) in India of its plasmid DNA-based vaccine for COVID-19, which if given a green light could become the first shot of its type to be cleared for widespread use in humans.  Leaving aside the possible technological milestone, the ZyCOV-D vaccine has a few characteristics that could […]

  • ICER revises its view of Aduhelm’s price – but not by much

    The Institute for Clinical and Economic Review (ICER) was very damning of Biogen’s new Alzheimer’s disease therapy Aduhelm when it issued its first report on the drug last month, and a swift update to its deliberations won’t be much comfort to the company. The cost-effectiveness watchdog has revised its calculation of a fair price range […]

  • ICER revises its view of Aduhelm’s price – but not by much

    The Institute for Clinical and Economic Review (ICER) was very damning of Biogen’s new Alzheimer’s disease therapy Aduhelm when it issued its first report on the drug last month, and a swift update to its deliberations won’t be much comfort to the company. The cost-effectiveness watchdog has revised its calculation of a fair price range […]

  • Turning words into actions with patient engagement

    Boehringer Ingelheim’s Carinne Brouillon tells us how the company co-created its recent Global Patient Partnership Summit with patients and how the meeting brought representatives together to design actionable projects for pharma-patient engagement. This article appears in our free digital magazine Deep Dive: Market Access 2021. Read a preview below: As pharma seeks new and better […]

  • Turning words into actions with patient engagement

    Boehringer Ingelheim’s Carinne Brouillon tells us how the company co-created its recent Global Patient Partnership Summit with patients and how the meeting brought representatives together to design actionable projects for pharma-patient engagement. This article appears in our free digital magazine Deep Dive: Market Access 2021. Read a preview below: As pharma seeks new and better […]

  • NICE backs Novartis’ Cosentyx for underdiagnosed back condition

    NICE has extended the range of indications that Novartis’ Cosentyx can be prescribed for by the NHS in England, giving a green light for its use in non-radiographic axial spondyloarthritis (nr-axSpA), a form of arthritis affecting the joints of the spine. IL-17 inhibitor Cosentyx (secukinumab) is already approved by NICE to treat ankylosing spondylitis or […]

  • Blueprint’s latest approval for Ayvakit sets up clash with Novartis

    Blueprint Medicines has claimed its second FDA approval for Ayvakit, getting the nod for advanced systemic mastocytosis (SM), a group of rare blood cancers currently treated mainly by Novartis’ Rydapt.  The FDA has cleared Ayvakit (avapritinib) for adults with SM, including the three major subtypes – aggressive disease with or without an associated haematological neoplasm, […]

  • Consumer group seeks resignation of top FDA staffers over Aduhelm okay

    With the first patient set to receive Biogen’s new Alzheimer’s drug Aduhelm in Rhode Island today, US consumer advocacy organisation Public Citizen is deeply unhappy about the FDA’s approval of the drug – and it wants the scalps of those responsible. In a letter to Secretary of Health and Human Services Xavier Becerra, the director […]

  • Artificial pancreas will be piloted in 1,000 diabetics, says NHS CEO

    NHS England will provide artificial pancreas devices to 1,000 type 1 diabetes patients as part of a pilot study of the technology, according to chief executive Sir Simon Stevens. The diabetics will be offered the closed-loop systems, which continually monitor glucose levels in the blood and uses the data to automatically adjust the dose of […]

  • A roadmap for adopting New Science 

    The pandemic’s disruption galvanised the biopharma industry into unprecedented action, demonstrating what’s possible when the global scientific community collaborates in exceptional ways and public-private partnerships drive innovation to address a common need. Can the biopharma industry step up again to shatter industry orthodoxy and create new pathways for innovation, access, and affordability? The adoption of […]

  • How the patient voice is becoming vital for drug approval

    Rebecca Sanders from Lipodystrophy UK tells us how the patient voice helped convince NICE to approve a much-needed drug for this rare disease, and explores how regulators and pharma companies can help make patient involvement in HTA more impactful. This article appears in our free digital magazine Deep Dive: Market Access 2021. Read below for […]

  • And then there were three: another FDA adcomm member quits over Aduhelm

    The fallout over the FDA’s decision to approved Biogen’s Alzheimer’s therapy Aduhelm continues to be felt, with a third member of its advisory committee resigning in protest.  The latest to depart the FDA’s Peripheral and Central Nervous System Drugs panel is Aaron Kesselheim of Harvard Medical School, joining fellow committee members Joel Perlmutter and David […]

  • Ocugen gets a black eye as FDA denies emergency OK for COVID jab

    Ocugen is the first company to be affected by the FDA’s recent decision to raise the bar on emergency use authorisations for COVID-19 medicines, and will have to file for full approval of its vaccine candidate. That will hold back the biotech’s Covaxin shot by several months, and the delay put pressure on Ocugen’s share […]

  • Double resignation over Aduhelm approval depletes FDA’s CNS adcomm

    Two members of an FDA advisory committee have now resigned in protest over Monday’s approval of Biogen’s Alzheimer’s disease drug Aduhelm despite an overwhelming vote against doing so. Washington University neurologist Joel Perlmutter was the first to announce he was stepping down from the Peripheral and Central Nervous System Drugs Advisory Committee on the day […]

  • Aduhelm approval could signal new era for CNS drugs; analysts

    The unexpected approval and lofty pricing of Biogen’s Alzheimer’s therapy Aduhelm is clearly the biopharma event of the year so far, but what are the broader implications for the industry as a whole? Analysts at RBC Capital Markets say that the decision points to an unprecedented shift in position for the FDA when it comes […]

  • FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

    The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. That was the damning conclusion of the influential Institute for Clinical and Economic Review (ICER) in the US to yesterday’s approval of Aduhelm (aducanumab), the first new treatment for Alzheimer’s […]

  • Scotland backs rare blood cancer drug Poteligeo rejected by NICE

    A north-south divide has emerged in UK medicines access after the Scottish Medicines Consortium (SMC) okayed Kyowa Kirin’s Poteligeo for two rare blood cancers, a few weeks after the drug was turned in England by NICE. Poteligeo (mogamulizumab) has been backed by the SMC for adults living with mycosis fungoides (MF) and Sézary syndrome (SS), […]

  • Jubilation for Biogen, Eisai as FDA says ‘yes’ to aducanumab

    Biogen and Eisai have defied the naysayers and secured an historic FDA approval for their amyloid-targeting antibody aducanumab as the first-ever disease-modifying treatment for Alzheimer’s disease. The accelerated approval from the US regulator has been given even though its own advisory committee comprehensively voted down the main study filed in support of the drug last […]

  • “A long way to go” for digital health reimbursement

    A recent analysis delves into the challenges companies face in getting digital health technologies reimbursed and presents recommendations for how these products can find a smoother path to market. This article appears in our free digital magazine Deep Dive: Market Access 2021. Read a preview below: In many ways, the healthcare sector is still wrestling […]

  • Time for a NICE change

    The National Institute for Health and Care Excellence (NICE) is world-renowned for their work assessing the clinical and cost-effectiveness of new treatments. In recognition of how the world has changed over its history, NICE is coming to the end of a period of review and consultation on their processes and methods. Leela Barham takes stock. […]

  • After FDA approval, Alkermes plans soft launch for antipsychotic Lybalvi

    The FDA approval of Alkermes’ new antipsychotic drug Lybalvi couldn’t have gone any better for the company, but it could still face a big challenge positioning the new drug in the highly genericised market. The US regulator approved Lybalvi (olanzapine/samidorphan) for both indications sought – schizophrenia and bipolar I disorder – with data on its […]

  • NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

    NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people […]

  • Janssen COVID jab cleared in UK as fears of third wave mount

    With concern already growing about a possible third wave of COVID-19 infections, the UK has cleared the use of a fourth vaccine from Johnson & Johnson’s Janssen division. The Medicines and Healthcare products Regulatory Agency (MHRA) gave a green light to the single-dose Ad26.COV2.S vaccine in people aged 18 and over by referencing the decision […]

  • GSK, Sanofi eye Q4 approval as COVID jab starts phase 3

    GlaxoSmithKline’s COVID-19 drive has advanced on two fronts, with the start of phase 3 trials of its Sanofi-partnered vaccine candidate as well as an FDA emergency approval for antibody sotrovimab, developed with Vir Biotech. GSK and Sanofi think they could be on track for approval of the recombinant protein-based vaccine – delivered with GSK’s immune-response […]

  • Rethinking access barriers to innovation

    Market access issues don’t stop once a product has passed HTA – and this is especially true for disruptive therapies like ATMPs. Experts from Executive Insight discuss how a holistic, multi-stakeholder approach can help companies overcome access barriers at all stages. This article appears in our digital magazine Deep Dive: Market Access 2021. Read on […]

  • Belgium halts dosing of J&J COVID vaccine in under 41s after death

    Belgium’s Ministry of Health has paused dosing of people under the age of 41 with Johnson & Johnson’s one-shot COVID-19 vaccine, following the death of a woman from what appeared to be a blood clot-related condition.  The unnamed woman – who was under the age of 40 – died on 21 May after being admitted […]

  • New Cell & Gene Therapy Collective seeks to boost UK adoption

    The UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – but there are still many barriers to wider adoption. These challenges often arise because these therapies go through the same value assessment processes as less complex medicines, and the infrastructure […]

  • AI meets RWE: The future of drug assessment?

    Savana’s Dr Ignacio Medrano explains how machine learning and natural language processing technology is opening up huge amounts of previously-inaccessible real-world evidence (RWE) for pharma and regulators. This article appears in out digital magazine Deep Dive: Market Access 2021. Read below for a preview: Real world evidence (RWE) is rapidly evolving. Having gone from being […]

  • CHMP backs bluebird bio’s gene therapy for rare disease ALD

    US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has given the go-ahead to bluebird’s therapy – known as Skysona (elivaldogene autotemcel) […]

  • Listening to ultra-rare disease communities

    When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global commercial biopharmaceutical company pioneering therapies for ultra-rare diseases, PTC Therapeutics is familiar with the challenges. The company prides itself on a patient-centric approach to treatment but there can be significant hurdles in patients accessing potentially life-changing treatments. […]

  • Japan approves two more COVID vaccines as fourth infection wave hits

    Japan has approved two more coronavirus vaccines, while expanding a state of emergency to cover the southern island of Okinawa. The two newly approved vaccines are from Moderna and AstraZeneca and will be added to a vaccine drive that began in mid-February using the Pfizer/BioNTech shot. However Reuters reported that the AstraZeneca vaccine will not […]

  • NICE rejects J&J’s prostate cancer drug Erleada in initial guidance

    NICE has rejected Johnson & Johnson’s oral prostate cancer drug Erleada (apalutamide) in patients with hormone-relapsed or hormone sensitive disease in first draft guidance. The cost-effectiveness body is assessing Erleada, plus androgen deprivation therapy (ADT), for prostate cancer in adults who have hormone-relapsed non-metastatic disease at high risk of metastasising and hormone-sensitive metastatic disease. Hormone […]

  • How to improve payer engagement with tailored value communication

    Pharmaceutical drug success rests on being able to achieve market access. Brands are facing increasing pressure to demonstrate strong value propositions in increasingly crowded therapeutic areas. However, value can mean different things to different payers. In this article, Cecilie Alstad, senior analyst at Research Partnership, discusses how an approach established in the world of marketing […]

  • Apellis set to take on Alexion as FDA clears PNH drug Empaveli

    Apellis Pharma has secured FDA approval for its complement C3 inhibitor Empaveli as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) – with a label that will allow it to challenge Alexion’s established therapies directly.  There had been speculation that the FDA might approve Empaveli (pegcetacoplan) only as a backup option for patients who don’t see […]

  • How will NICE fare in a post-Brexit world?

    NICE has a global reputation as a pioneering HTA – but is that influence at risk now that the UK has left the EU? Experts from ICON give us their views on the past, present and future of NICE’s standing on the world stage. This article appears in our digital magazine, Deep Dive: Market Access […]

  • NICE says final ‘no’ to BMS’ Zeposia for multiple sclerosis

    NICE has issued a final “no” to Bristol-Myers Squibb’s oral MS drug Zeposia (ozanimod), in a decision that prevents access for patients in England and Wales. This ruling means that there will be a divide in access to Zeposia in the UK, after the Scottish Medicines Consortium allowed funding in February for the drug in […]

  • Research predicts the post-COVID landscape for HCP engagement

    IQVIA’s Liz Murray takes us through the company’s latest research into HCPs’ attitudes to pharma engagement, and asks how pharma sales forces can adapt for future changes. This article appears in full in Deep Dive: Market Access 2021. Read a preview below: Recent ChannelDynamics data from IQVIA shows that while there has been a marked […]

  • Alvotech files lawsuit challenging AbbVie’s Humira patent thicket

    Biosimilars firm Alvotech has filed a lawsuit that challenges AbbVie’s legal defences surrounding the inflammatory disease drug Humira in the US. Alvotech is challenging the patent thicket surrounding Humira, a common ploy in pharma that seems to be particularly effective in the US. Several biological drugs have remained unchallenged on the US market thanks to […]

  • NICE says ‘no’ to Pfizer’s Vyndaqel for rare heart condition

    NICE has rejected regular NHS funding for Pfizer’s Vyndaqel (tafamidis) for a rare heart condition, although it has left the door open for negotiations if the pharma decides to drop its price. Pfizer had appealed against draft guidance from NICE that rejected Vyndaqel for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) on cost grounds. The pharma unsuccessfully […]

  • FDA authorises Pfizer/BioNTech vaccine for adolescents

    The FDA has authorised Pfizer/BioNTech’s vaccine for 12- to 15 year-olds under emergency rules. In a statement the regulator said it had expanded the vaccine’s Emergency Use Authorization to include the new age group. The vaccine was first approved under the emergency protocols on 11th December, for people aged 16 years of age and older. […]

  • Scotland backs NHS use of Alexion’s Ultomiris for rare disease aHUS

    NHS patients in Scotland with the rare disease atypical haemolytic uremic syndrome (aHUS) will now be able to access treatment with Alexion’s Ultomiris, ahead of their counterparts in England and Wales.  aHUS is an ultra-rare disease that can cause progressive injury to the kidneys via damage to the walls of blood vessels and blood clots […]

  • A year on: pandemic driven trends in HCP engagement

    IQVIA’s John Procter explores why the industry should be paying attention to the changing HCP customer experience in the wake of COVID-19. This article appears in full in this month’s Deep Dive digital magazine on Market Access. Here is a preview: As we reach the end of the first quarter of 2021, our customers continue […]

  • EU starts rolling review of GSK’s antibody for COVID-19

    The EMA has begun a rolling review of a COVID-19 antibody developed by GlaxoSmithKline and Vir BioTech which could become the fourth drug of its type cleared for early use in the EU.  Sotrovimab (VIR-7831) – also known as GSK4182136 – is being assessed as a treatment for patients with SARS-CoV-2 infection aged 12 and […]

  • Patients in England get fast access to AZ’s Tagrisso in early lung cancer

    The UK’s drugs regulator has swiftly granted a licence extension for AstraZeneca’s Tagrisso in certain patients with early stage lung cancer, with NICE allowing fast access to patients on England’s NHS. The arrangements under Project Orbis, a global project run by the FDA that the Medicines and Healthcare products Regulatory Authority is supporting, aim to […]

  • Pfizer shares slump as support grows for COVID-19 vaccine patent waiver

    Shares in COVID-19 vaccine manufacturers have taken a hit after the US signalled its support for waiving patents on them, a moved aimed to boost global supplies of the life-saving shots. Waiving the patent on proprietary knowledge would allow it to be shared with other manufacturers and the matter is being discussed by the World […]

  • Hidden compliance risks for life sciences companies

    The life sciences history industry has some unique challenges when it comes to compliance. But if you think third party compliance risks are bad, don’t take your eye off fourth and fifth parties, says Allan Matheson. Early life science pioneers in foreign markets earned a reputation for lax ethical behaviour, making them a prime target […]

  • AstraZeneca’s Farxiga claims first SGLT2 kidney disease OK from FDA

    AstraZeneca is already seeing sales rocket for its SGLT2 inhibitor Farxiga in heart failure, and is expected to gather even more momentum after claiming FDA approval in chronic kidney disease (CKD).  The US regulator has cleared Farxiga (dapagliflozin) to reduce the risk of kidney damage, end-stage kidney disease, cardiovascular, death and hospitalisation for heart failure […]

  • Galapagos UK’s Michael Smyth on RNA-based tech: the pharmaphorum podcast

    Episode 35 of the pharmaphorum podcast hears from Michael Smyth about Galapagos UK and joining the company as medical director just as COVID-19 hit. With RNA-based technology hitting the headlines in recent months due to its use in Covid vaccines, he also discusses the version of it that Galapagos uses and how it can produce […]

  • Deep Dive: Market Access

    It’s fair to say this is the most interesting time ever for market access. Not only has COVID forced companies, governments and healthcare systems to work towards approving drugs and vaccines in record times, the sector is also facing an influx of digital therapeutics and advanced drugs that don’t fit neatly into existing access frameworks. […]

  • Indivior pays $300m to settle Suboxone marketing allegations

    Indivior has paid out $300 million to resolve civil claims from US states after it was last year found guilty against criminal charges of improper marketing of the opioid addiction drug Suboxone. Last year, Indivior reached a $600 million settlement plea deal to settle criminal fraud charges, after US authorities found the company’s conduct had […]

  • UK orders 60m ‘booster’ doses of Pfizer/BioNTech COVID vaccine

    The UK has ordered an extra 60 million doses of the Pfizer/BioNTech COVID-19 vaccine, which could be used as a potential booster campaign in the autumn. Health secretary Matt Hancock made the announcement in a press briefing, revealing that officials think they may have to boost protection for the population during the winter. The UK […]

  • FDA sets September review date for Calliditas’ rare kidney disease drug

    Calliditas Therapeutics could have its first product on the market in the US this autumn, as the FDA has now started a priority review of Nefecon for rare disease primary IgA nephropathy (IgAN).  The Swedish biotech says the IUS regulator has set an action date of 15 September for its review of Nefecon, a novel […]

  • European Commission legal battle with AZ over COVD vaccines contract

    The European Commission has officially begun legal proceedings against AstraZeneca over supplies of COVID vaccines. The legal action marks an escalation in the dispute between the EU and AZ over the supply of coronavirus vaccines. Under pressure to roll out vaccinations, the European Commission has accused AZ of failing to live up to a contract […]

  • CHMP backs Roche’s Enspryng for rare nerve disease NMOSD

    Roche’s Enspryng has been recommended for approval in the EU for treating neuromyelitis optica spectrum disorder (NMOSD), extending the treatment options for people with the life-threatening rare disease.  The CHMP has backed the drug in patients aged 12 or more with NMOSD that tests positive for aquaporin-4 (AQP4) antibodies, a biomarker seen in around 80% […]

  • Calliditas eyes 2022 approval in EU for rare kidney disease drug Nefecon

    Calliditas Therapeutics is on course to launch its first product, Nefecon for rare disease primary IgA nephropathy (IgAN), in the first half of 2022 after getting the green light for an accelerated review by the EMA.  The Swedish biotech is planning to file Nefecon for approval in the second quarter to the EU regulator, which […]

  • Jazz lifted by third EU approval for GW Pharma’s Epidyolex

    GW Pharmaceuticals’ Epidyolex has been approved in the EU for a third epilepsy indication, giving a boost for the company ahead of its $7.2 billion takeover by Jazz Pharma.  The cannabis-derived drug has been cleared by the EMA to treat seizures associated with tuberous sclerosis complex (TSC), a condition in which mostly benign tumours grow […]

  • FDA pens stinging report on Emergent COVID vaccine plant

    Emergent BioSolutions’ difficult month has been made worse by an FDA report into its facility in Baltimore, which has been blamed for the wastage of millions of doses of Johnson & Johnson’s COVID-19 vaccine.  The US regulator says it completed an inspection of the Bayview plant run by the contract manufacturing organisation (CMO) which uncovered […]

  • EU safety regulators clear J&J COVID shot despite tiny clot risk

    The benefits of Johnson & Johnson’s COVID-19 vaccine outweigh a tiny risk of unusual blood clots forming, Europe’s drugs regulator has said. In a decision that will allow the rollout of vaccines in certain EU states, the European Medicines Agency’s safety committee said there appears to be a link to unusual blood clots combined with […]

  • Hikma shares rally after US launch of Advair generic resumes

    Shares in Hikma have rallied as it resumed the launch of its generic Advair Diskus in the US, after clearing up a final piece of bureaucracy with the FDA. The London-listed firm is one of several companies that had been trying to make generics of GlaxoSmithKline’s former respiratory diseases blockbuster for the US market. Advair […]

  • Enhertu cleared for NHS use via Cancer Drugs Fund

    NICE has recommended the use of AstraZeneca and Daiichi Sankyo’s Enhertu in draft guidance, the first time the drug has been commissioned in any European country.  The UK health technology assessment (HTA) agency has backed Enhertu (trastuzumab deruxtecan) as a therapy for HER2 positive breast cancer which can’t be surgically removed or which has spread […]

  • NICE unveils five year plan promising faster access to medicines

    NICE has included proposals to speed up evaluations and focus on new technology such as digital health in a new strategy to provide faster access to new medical treatments and innovations. The cost-effectiveness body has produced a new vision for the next five years, after reflecting on lessons learned during the COVID-19 pandemic. NICE said […]

  • NICE unveils five year plan promising faster access to medicines

    NICE has included proposals to speed up evaluations and focus on new technology such as digital health in a new strategy to provide faster access to new medical treatments and innovations.  The cost-effectiveness body has produced a new vision for the next five years, after reflecting on lessons learned during the COVID-19 pandemic. NICE said […]

  • EMA starts review of GSK/Vir COVID-19 antibody

    The EMA has started reviewing the emergency use application for GlaxoSmithKline and Vir Biotech’s COVID-19 antibody VIR-7831, which could become the fourth drug of its type cleared for early use in the EU.  The monoclonal antibody – also known as GSK4182136 – is being evaluated for the treatment of people with mild or moderate COVID-19 […]

  • World Orphan Drug Congress USA Virtual 2021

    On Wednesday, April 28th, the World Orphan Drug Congress USA is being held online for free! The conference brings together pharma, biotechs, government, payers, investors and patient/patient advocates in the rare disease space to discuss the latest developments that will lead to the future of orphan drug development. We are thrilled to announce some amazing speakers […]

  • Three pillars of post-pandemic launch excellence

    IQVIA’s Sarah Rickwood reviews the launch of innovative medicines in 2020 and outlines three key pillars of activity for companies to focus on and address in 2021. It is undeniable that 2020 was a particularly challenging year to launch non-COVID innovative prescription medicines. The launch environment was directly impacted by the COVID-19 pandemic, which hit […]

  • NICE recommends Alexion’s long-acting Ultomiris for PNH

    NICE has recommended Alexion’s long-acting Ultomiris (ravulizumab) for the rare disease paroxysmal nocturnal haemoglobinuria (PNH) in final draft guidance. Ultomiris is Alexion’s follow-up to its rare diseases blockbuster Soliris (eculizumab), which allows for  an eight-week dosing schedule after a loading phase. This is more patient-friendly than Soliris, which requires infusions every two weeks, a considerable […]

  • Czech Republic swoops for Denmark’s 2.4m unwanted AZ COVID shots

    The Czech Republic wants to buy around 2.4 million doses of the AstraZeneca/Oxford University vaccine ditched by Denmark, which has said it will stop using it because of concerns about rare blood clotting side-effects. Czech deputy prime minister Jan Hamacek said on twitter that he had instructed the country’s ambassador in Denmark to try to […]

  • Global impact: How regulatory affairs is shaping development of cutting-edge pharma innovation

    When it comes to developing cutting-edge innovations, Astellas aims to be at the forefront. The pharma organisation prides itself on having an open, collaborative, “science first” approach to help ensure breakthrough discoveries have the very best chance of long-term success in bringing new treatment options to patients worldwide. “Our mission is to turn innovative science […]

  • England begins Moderna vaccine rollout as lockdown eases

    England is beginning its rollout of the Moderna COVID-19 vaccine, offering an alternative to the AstraZeneca/Oxford University jab following a potential link to a very rare kind of blood clot. The Moderna shot is the third vaccine to become available in the country, which has made considerable progress with its inoculation campaign. Its arrival is […]

  • Scotland opens door to Translarna for Duchenne muscular dystrophy

    A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics’ Translarna for the first time.  The Scottish Medicines Consortium (SMC) says that Translarna (ataluren) can be provided by NHS Scotland for a period of three years, once PTC […]

  • Provention faces delay in FDA review of diabetes prevention antibody

    The FDA says it has uncovered “deficiencies” in the marketing application for Provention Bio’s much-anticipated drug teplizumab for the prevention of type 1 diabetes (T1D) that could delay its review. The issues are holding up discussions between the agency and company about labeling and post-marketing requirements for teplizumab, and are concentrated around the pharmacokinetics data […]

  • Gilead scores US approval for breast cancer drug that wowed ESMO

    Gilead’s $21 billion purchase of Immunomedics and its potential blockbuster Trodelvy continues to pay off, as the drug has now been approved by the FDA for triple-negative breast cancer.  Trodelvy (sacituzumab govitecan) was granted accelerated approval by the FDA in April last year, and is now indicated for adults with triple-negative breast cancer who have […]

  • Acadia puts blame on FDA as Nuplazid rejected for dementia

    Acadia Pharmaceuticals is butting heads with US regulators again, this time over the FDA’s decision to reject its Nuplazid (pimavanserin) for use in dementia-related psychosis (DRP) – with the company bemused by the agency’s apparent pivot on an agreed trial design.  The FDA has rejected the expansion of the drug – originally approved to treat […]

  • NICE backs Sobi’s Kineret for rare inflammatory disease

    The UK’s cost-effectiveness agency NICE has recommended that Sobi’s Kineret can be used to treat NHS patients with Still’s disease, a rare form of arthritis affecting children and adults. The final guidance concludes that interleukin-1 receptor antagonist Kineret (anakinra) can be used as a treatment for two forms of Still’s, namely systemic juvenile idiopathic arthritis […]

  • World leaders call for international pandemic preparedness treaty

    More than 20 world leaders have called for an international treaty for pandemic preparedness to protect the world from future health crises. Backed by the World Health Organization, the treaty would “dispel the temptations of isolationism and nationalism” in the event of another crisis such as COVID-19. In a commentary backed by the 24 leaders, […]

  • Moderna COVID-19 shot available in UK next month

    The Moderna COVID-19 vaccine will be added to the UK’s arsenal of coronavirus vaccines in April, when the country will focus on handing out second doses to those already vaccinated. Minister for vaccine deployment Nadhim Zahawi said that supplies of the Moderna shot will become available during a near-pause of the first dose programme. The […]

  • CHMP backs Celltrion’s regdanvimab antibody for COVID-19

    The EU looks set to approve emergency use of a third antibody therapy for COVID-19 after its human medicines committee backed use of Celltrion’s regdanvimab at its March meeting.  Regdanvimab (CT-P59) – which is currently under a rolling review at the EMA – can be used for the treatment of COVID-19 in adults who don’t […]

  • The Brexit deal; shot in the arm or kick in the teeth for the pharma sector?

    The UK Government and EU Commission trumpeted their Brexit trade deal, struck at the end of December, as ‘comprehensive’, the ‘biggest yet’.  But a closer inspection of the EU-UK Trade and Cooperation Agreement (TCA) renders these statements largely illusory for the pharma sector. While pharma companies grapple with the effects of Brexit, there are undoubtedly […]

  • EU stops short of vaccine export controls after summit

    The European Union has stopped short of export controls on vaccines after leaders met at a summit to discuss the issue of shortfalls in supply. At the meeting of the European Council leaders said that toughening export controls was an option but a post-summit statement emphasised the importance of global supply chains to produce vaccines. […]

  • Boris Johnson slammed for saying “greed” is behind UK vaccine success

    Boris Johnson’s sense of humour seems to have got him into trouble again, at the most delicate of times. The Prime Minister reportedly told Conservative backbench MPs on a Zoom call that “capitalism” and “greed” was behind the success of the UK’s COVID-19 vaccination programme, before backtracking after realising he had made a blunder and […]

  • NICE backs ovarian cancer and multiple myeloma combination drugs

    NICE has recommended funding for two cancer drug combinations for ovarian cancer and multiple myeloma. In final draft guidance that applies to England, NICE said AstraZeneca/MSD’s Lynparza (olaparib) plus Roche’s Avastin (bevacizumab) should be reimbursed by the Cancer Drugs Fund in people with ovarian cancer that have responded to platinum chemotherapy. Early results from the […]

  • NICE backs ovarian cancer and multiple myeloma combination drugs

    NICE has recommended funding for two cancer drug combinations for ovarian cancer and multiple myeloma. In final draft guidance that applies to England, NICE said AstraZeneca/MSD’s Lynparza (olaparib) plus Roche’s Avastin (bevacizumab) should be reimbursed by the Cancer Drugs Fund in people with ovarian cancer that have responded to platinum chemotherapy. Early results from the […]

  • Novartis says £1.8m Zolgensma is model for gene therapy pricing

    Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Muscular Atrophy (SMA).  Zolgensma (onasemnogene abeparvovec) has a list price of £1,795,000 excluding VAT in the UK, but Novartis says the price is justified by […]

  • Life sciences must become cloud-first to seize the science of tomorrow

    If the power of the life sciences industry to innovate and collaborate were ever in doubt, the speed at which vaccines for COVID-19 were discovered, developed, trialled, approved and manufactured have confirmed just how dynamic the industry can be. And that dynamism, innovation and collaboration are all qualities that the industry must continue to nurture […]