Sobi gets EU nod for Doptelet in rare blood disorder ITP

Sweden’s Sobi has secured EU approval for Doptelet in primary chronic immune thrombocytopenia (ITP), an indication that the company expects to accelerate sales of the drug.

Doptelet (avatrombopag) – an oral thrombopoietin receptor agonist – has been cleared to boost depleted levels of platelets in the blood of patients with ITP, an autoimmune clotting disorder that causes excessive bruising and bleeding.

The drug was approved last year for the treatment of low platelets in adults with chronic liver disease (CLD) before surgery, a fairly small indication that so far has allowed the drug to bring in just a few million dollars in revenue per quarter.

Sobi acquired Doptelet via its $915 million takeover of Dova Pharmaceuticals in 2019, adding to its haematology and rare disease franchise. ITP affects around 100 people per million worldwide.

The European approval of Doptelet as a second-line treatment for ITP after therapies such as corticosteroids and immunoglobulin drugs comes after a green light by the FDA in July 2019. According to some analysts it could catapult sales to several hundred million dollars a year, perhaps even broaching the $1 billion threshold.

Sobi’s drug will have to compete with various drugs including Novartis’ Promacta (eltrombopag) – which made $1.2 billion in revenues in the first nine months of 2020 – as well as Rigel Pharma/Grifols’ recently-approved oral drug Tavalisse/Tavlesse (fostamatinib) and Amgen’s injectable Nplate (romiplostim) in the ITP indication.

Doptelet is claimed to have a cleaner clinical profile compared to Promacta, lacking the dietary restrictions, drug interactions and liver side effects seen with Novartis’ drug, giving it a chance to become the market leader – although its initial US rollout has been affected by the coronavirus pandemic.

Sobi claimed the drug had captured a 5% share of the US market for ITP therapies in its third-quarter 2020 results statement, bringing in sales of SEK 145 million (around $17 million) in the three-month period, a rise of 58% on the same period of 2019.

Prospects for Sobi’s avatrombopag franchise took a big knock last October however when the drug failed a phase 3 trial as a treatment for chemotherapy-induced thrombocytopenia (CIT), which has no approved therapies.

CIT is a common complication of cancer treatment so success there could have unlocked blockbuster sales for Doptelet. However while Sobi’s drug increased platelet counts relative to placebo, it wasn’t able to reduce the need for platelet transfusions or chemotherapy dose reductions, the study’s main objective.

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Tessa’s CD30 CAR-T Therapy Receives EMA’s PRIME Designation for Relapsed or Refractory Classical Hodgkin Lymphoma


  • The PRIME designation is based on two Phase I/II studies in R/R cHL conducted at Baylor College of Medicine and the University of North Carolina Lineberger Comprehensive Cancer Center
  • The studies showed the complete disappearance of tumors in ~60% of patients at the highest dose level with no serious toxicities, associated with several other CAR-T therapies. The results were published in Clinical Oncology
  • The company plans to commence a multi-center pivotal study in the US during 2021. Additionally, P-I clinical study for patients with r/r CD30 positive NHL is open for enrollment in the US

Click here ­to­ read full press release/ article | Ref: PR Newswire | Image: PR Newswire

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ViiV Healthcare’s Tivicay (dolutegravir, dispersible tablets) Receives the EMA’s Approval for Children with HIV


  • The approval is based on P1093 & ODYSSEY (Penta20) studies assessing safety, tolerability & dose-finding of Tivicay (5mg) in pediatric patients aged 4wks.-18yrs. while the second study assessed the 1L & 2L treatment in patients of the same age
  • The approval includes updated dosing recommendations for Tivicay film-coated tablets (10/25/50mg) for children aged ≥6yrs. and weighing at least 14kg, bringing these in line with the WHO weight bands
  • Dolutegravir is the first integrase inhibitor used in combination with other antiretroviral agents for the treatment of HIV-1 infection in pediatric patients (treatment-naïve or -experienced but INSTI- naïve) aged at least 4wks. and weighing at least 3kg.

Click here ­to­ read full press release/ article | Ref: VIIV Healthcare | Image: Smart Industry News

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EMA says AstraZeneca’s COVID-19 shot could be approved on 29 January

The European medicines Agency (EMA) said this morning it has received a marketing application from AstraZeneca for its COVID-19 vaccine, already rolling out in the UK, and could give it the go-ahead later this month.

The filing for conditional marketing approval is scheduled for review by the EMA’s CHMP human medicine committee at a meeting on 29 January, and if all goes well it could be authorised on that day, according to the regulator.

The European Commission will then fast-track its decision-making process, says the EMA, with a view to granting a conditional marketing authorisation “within days”, a timeframe which was welcomed by Commission President Ursula von der Leyen.

AZ’s AZD1222 shot – which was developed with Oxford University – was cleared by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on 30 December, and since then it has also been given emergency approval in several other countries including India.

The filing comes as the EU is facing criticism for the slow roll-out of its coronavirus vaccination programme as infection rates soar in the 27 member states.

While individual EU countries make the decisions about who to vaccinate, the Commission is coordinating the acquisition and allocation of supplies, and there have been complaints the process is taking too long.

The US and Britain have both vaccinated 1%-2% of their populations, according to an Economist report citing figures from the Our World in Data website, while Israel is leading the field at 16%. In contrast, Germany has managed just 0.4%, France didn’t cross the 1,000 threshold until 4 January, and the Netherlands only started vaccinating until 6 January.

So far the EMA has conditionally approved two coronavirus vaccines – Pfizer/BioNTech’s Comirnaty last month and Moderna’s candidate last week – and swift approval of the AZ vaccine should allow an acceleration in vaccination rates in the EU.

As it stands, the UK has vaccinated more people than the entirety of the EU combined, with the latest government figures indicating 2.3 million people have now received the first of two required doses, saying it plans to immunise all adults in the country by the autumn.

So far the effect of vaccination is imperceptible, however, as the UK recorded more than 46,000 new cases of COVID-19 yesterday, and 529 deaths, with NHS capacity creaking under the weight of over 32,000 people hospitalised with the infection.

The EU has 400 million doses of the AZ vaccine on order, part of a procurement programme that so far extends to 2.3 billion doses.

Last week the Commission said it intended to order an additional 200 million doses of the BioNTech/Pfizer, with the option to acquire another 100 million doses, taking its total to 600 million doses.

It has also agreed deals for the supply of 160 million doses of the Moderna shot, 400 million apiece for candidates in testing at Johnson & Johnson and CureVac, and 300 million of a Sanofi/GlaxoSmithKline candidate that has been delayed by clinical trial snags.

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EMA will use Panalgo software for real-world data analytics

Six months after rebranding from BHE, Panalgo has won a contract to supply the European Medicines Agency (EMA) with its IHD data analytics platform, pledging to streamline its public health efforts.

IHD – or Instant Health Data – will be used by the EMA to carry out data analyses and examine medicinal product utilisation, answer questions about safety and efficacy, and understand how treatments perform in real-world settings, said the Boston, US-based company.

The use of real-world evidence (RWE) has grown rapidly in pharma over the last few years, as healthcare payers and regulatory agencies have tried to get a deeper understanding of the impact medicinal products have in actual practice, rather than just within clinical trial settings.

“Our company is strongly aligned with the EMA’s public health goals, for which timely evidence-based insights are of particular importance, especially during this COVID-19 pandemic,” commented the US company’s CEO Joseph Menzin.

Panalgo is also opening an office in Amsterdam, the home of the EMA, in order to provide close support for the roll-out and management of the new software at the regulator.

Panalgo says IHD’s library of healthcare-specific algorithms does away with the need for complex programming and allows users to “focus on what matters most: turning data into insights”, using sources like electronic health records and patient registries.

There has been a spike in the use of data analytics by healthcare organisations during the coronavirus pandemic to try to guide the public health response to the crisis, for example to make effective, real-time use of resources.

The new agreement with the EMA adds a new dimension to Panalgo’s data analytics business, as most of its clients have so far been life science companies and research organisations.

The company says IHD passed a rigorous assessment by the EMA – in competition with software from other providers – and will support decision-making at the population level, including therapy risk/benefit and life cycle analyses that can be used in public health planning.

“Most of Panalgo’s life sciences clients already rely on IHD to perform rapid analyses that support regulatory initiatives,” said Menzin.

“The IHD platform will be a valuable tool to allow collaboration between drug manufacturers and regulatory agencies, such as the EMA,” he added.

Image by Ceescamel – Own work, via Wikimedia, CC BY-SA 4.0

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Elsevier to Work with Heel for Mechanism-based Drug Action Discovery

Elsevier, the data analytics business specialized
in science and health, and Heel, a pharmaceutical company specialized in
developing and manufacturing medicines made from natural ingredients, have
recently completed a series of research projects with a focus on improving
exploratory preclinical studies.

“We at Heel are pioneers in the field
of systems research and have a strong commitment to scientific excellence and
the generation of evidence. Our aim is to find out how these medicines work in
the body and to develop therapies which are tailored even more to patients’
medical needs,’’ said Dr. Kathrin Hemmer, a scientist at Heel. “We chose to
partner with Elsevier because of its expertise in scientific information search.
Assistance from the Professional Services group allowed us to get a single
access to all the Elsevier’s R&D solutions advancing our exploratory
research programs.”

“Research success requires connecting, combining
and harmonizing data from different sources. We are helping researchers to select
the best evidence-based strategy for mechanism-based drug action”, said Dr.
Taisiya Bezhaeva, Professional Services Consultant at Elsevier.

“Together with Heel, we designed a
series of projects to find preclinical models for drug action discovery,
identify key biomarkers, and research platforms validated by the international
research community. We focused on the broad range of disease areas, overall
covering more than 10000 literature sources, as well as FDA and EMA drug
approval documents. We also supported researchers by providing key opinion leaders
(KOL) and potential academic and commercial partners helping Heel to direct and
facilitate the course of their studies”, said Dr. Maria Shkrob, Senior Consultant
in Professional Services at Elsevier.  



  • Connecting, combining and harmonizing data from different
  • Big-data & evidence-based approach to identify complex molecular
    mechanisms and biological networks for natural active compounds
  • Such approach combined with advanced text-mining technologies and
    statistics is a powerful, feasible
    and universal analytic method to select the best strategy for exploratory
    research to demonstrate pharmacodynamic actions, safety and efficacy
  • Support to strengthen scientific credibility and
    facilitate market positioning

Novo Nordisk Reports Submission of Label Extension Application to the EMA for Semaglutide (once weekly, 2.0 mg) for T2D


  • The submission is based on SUSTAIN FORTE trial assessing Ozempic (2.0mg, qw) vs Ozempic (1.0mg) in 961 people with T2D in need of treatment intensification
  • Result: 2.0 mg of dose achieved significant and superior reduction in HbA1c @40wks., both the doses are safe and well-tolerated
  • Ozempic is a glucagon-like peptide-1 (GLP-1) analogue and is currently approved in the EU for 0.5 mg and 1.0 mg doses to treat T2D in adults

Click here ­to­ read full press release/ article | Ref: GlobeNewswire | Image: Facebook

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Janssen Report MAA Submission of Amivantamab to EMA for Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations


  • The EMA Submission is based on P-l CHRYSALIS study to evaluate the safety and efficacy of Amivantamab as a monotherapy and in combination with Lazertinib in adult patients for the treatment of advanced NSCLC
  • Result of CHRYSALIS Study assessed efficacy using overall response rate per Response Evaluation Criteria, CBR, DOR and PFS as well as the safety profile of amivantamab
  • Amivantamab is an investigational, fully – human EGFR-MET bispecific antibody with immune cell – directing activity that targets tumours with activating and resistance EGFR mutations and MET mutations and amplifications

Click here ­to­ read full press release/ article | Ref: Janssen | Image:

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Daiichi Sankyo, AZ close on EU approval of Enhertu for breast cancer

Daiichi Sankyo and AstraZeneca could be just weeks away from an EU approval for their antibody-drug conjugate (ADC) for breast cancer – Enhertu – which is tipped to become a multibillion-dollar blockbuster.

At its meeting last week, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Enhertu (trastuzumab deruxtecan) for patients with unresectable or metastatic HER2-positive breast cancer, who have been previously treated with other anti-HER2 drugs.

Enhertu has already been approved for third-line use in HER2-positive breast cancer in the US at the end of 2019 and in Japan earlier this year, based on the results of the phase 2 DESTINY-Breast01 trial in 184 patients, which revealed that the drug shrank tumours in 61% of recipients.

Revenues from the drug  in the first nine months of 2020 came in at $136 million – including $60 million in the third quarter.

Sales were recorded by Daiichi Sankyo, with AZ pocketing $63 million in profit sharing, and according to AZ the drug is now the most prescribed medicine in the third-line and fourth-line settings of HER2-positive metastatic breast cancer.

Enhertu consists of the antibody used in Roche’s blockbuster HER2 antibody Herceptin (trastuzumab), linked to a topoisomerase inhibitor that is toxic to cancer cells. Around one in five patients with breast cancer are considered HER2 positive, which is associated with aggressive disease, a high recurrence rate, and an increased risk of dying.

It works by latching on to HER2-positive cancer cells and delivering a payload to kill them, while ignoring healthy cells, in patients who have failed to respond to Roche’s HER2-targeting cancer drugs Herceptin, Perjeta (pertuzumab), and ADC Kadcyla (trastuzumab emtansine).

Kadcyla was once tipped to become the go-to treatment HER2-posiitve breast cancer when first line drugs like Herceptin/Perjeta had failed, but failed to meet the mark in pivotal trials, truncating its sales growth although it still managed to break into the $1 billion-plus bracket.

Daiichi Sankyo is confident Enhertu can top Roche’s ADC, and also expand the use of HER2 drugs into new cancers like HER2-positive gastric cancer – an indication that is under review by the FDA with a verdict due early next year – and HER2-positive non-small cell lung cancer (NSCLC).

The intention is to gradually position the drug for earlier-line use in breast, gastric and lung cancer, and eventually to try to expand its use into certain low HER2-expressing tumours.

If all the pieces fall into place it reckons peak sales could reach $4.5 billion, and there are plenty of analysts predicting that the drug could quickly breach the $2 billion-a-year threshold.

AZ’s confidence in the potential of Enhertu is evidence from the terms of its late 2019 licensing deal with Daiichi Sankyo, which included a hefty $1.35 billion upfronting a deal that could be worth up to $6.9 billion if all the ADC’s development and sales objectives are achieved.

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Cyberattack targets EMA, hacks COVID-19 vaccine data

The European Medicines Agency (EMA) says it suffered a cyberattack, with documents relating to a Pfizer and BioNTech’s COVID-19 vaccine accessed.

In a terse statement, the EU regulator confirmed its security had been breached and said it had launched an investigation with law enforcement, but would not be providing any additional information while that probe was underway.

Shortly after however BioNTech confirmed that documents submitted as part of its marketing application for coronavirus vaccine BNT-162b had been accessed by the hackers.

Responding to fears that the review could be delayed, the company said it had been assured by the EMA that the timeline should not be affected. The agency has indicated it should complete its review by 29 December.

BNT-162b is already approved in the UK and Canada, and the first UK patients started to receive the shot on Tuesday this week. The EMA is also reviewing another vaccine from Moderna, but at the moment it’s not clear if data from that programme has also been compromised.

“It is important to note that no BioNTech or Pfizer systems have been breached in connection with this incident and we are unaware that any study participants have been identified through the data being accessed,” said BioNTech in a statement on its website.

It added that it had publicised the breach “given the critical public health considerations and the importance of transparency”.

The cyberattack came just days after international enforcement agency Interpol warned that organised criminals may try to target COVID-19 vaccine supply chains, for example by falsification, theft and illegal advertising of unlicensed shots.

Europol meanwhile warned earlier this year that criminal networks are exploiting the COVID-19 pandemic with a surge in cybercrime, targeted thefts and counterfeiting, including attempts to target organisations through business email compromise (BEC), which can be used to harvest sensitive data, siphon off funds or damage its reputation.

There’s no indication yet who was behind the EMA hack, but a volunteer group set up to tackle cybercrime related to COVID-19 – CTI League – has suggested that one motivation could be to uncover details about the supply and distribution of vaccines.

The group’s found Marc Rogers told Reuters that information “potentially significantly increases the attack surface for the vaccine”.

IBM recently said an email phishing campaign had targeted organisations linked to the Cold Chain Equipment Optimisation Platform (CCEOP) of Gavi, the international vaccine alliance, suggesting that the sophistication of the assault pointed to a nation state being the culprit.

“Without a clear path to a cash-out, cyber-criminals are unlikely to devote the time and resources required to execute such a calculated operation,” according to the tech giant’s Security X-Force.

There has also been reports that hackers linked to North Korea, South Korea, Iran, Vietnam, China and Russia have tried to steal information about vaccines, targeting pharma companies and other organisations involved in COVID-19 medicine R&D, according to the news agency.

In October, Indian pharma company Dr Reddy’s Laboratories, which is helping to conduct late-stage testing of Russia’s Sputnik V COVID-19 vaccine, said it had been hit by a cyberattack that disrupted its production facilities.

Sam Curry, chief security officer at Cybereason, is convinced that a nation state is behind the EMA attack, saying: “Cyberattacks on the global COVID-19 vaccine distribution network from nation-states China, Russia and North Korea are diabolical in nature and acts of war.”

While acknowledging the average person “might be asking themselves why nation-state actors…are deliberately sowing doubt and confusion around the world at the worst possible time,” Curry says there is tremendous value in interfering with the distribution of COVID-19 vaccines.

“A COVID-19 vaccine is a strategically valuable asset to nation-states; whoever gets a vaccine distributed first has an economic advantage. It is the ultimate IP with immediate value. It is like having an oil rush, a data advantage or territorial gain in older real political terms.”

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BioNTech and Pfizer Report CMA Submission of BNT162b2 to EMA for COVID-19


  • The companies have submitted CMA to the EMA for BNT162b2, against COVID-19. The submission completes the rolling review process initiated on Oct 6, 2020
  • The submitted data showed a 95% efficacy rate, efficacy was consistent across age, gender, race, and ethnicity demographics, with an observed efficacy in adults aged ≥65yrs. of >94%, favorable tolerability with no safety concerns
  • In addition to submission to EMA, FDA & MHRA, the companies have also initiated additional rolling submissions across the globe including in Australia, Canada, and Japan, and plan to submit applications to other regulatory agencies globally

Click here ­to­ read full press release/ article | Ref: Globe Newswire | Image: Stat

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Vertex gets EU okay to treat younger cystic fibrosis patients

A new EMA approval has expanded the use of Vertex Pharma’s exon-skipping cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations.

The regulator has given a green light to use of Symkevi (tezacaftor/ivacaftor) with Vertex’ Kalydeco (ivacaftor) in patients ages six years and older who have two copies of the F508del mutation in the CFTR gene, or one F508del copy and one of 14 other so-called “minimal function” mutations in CFTR.

The new EU approval comes after the FDA approved the expanded label last year, and provides the first treatment option that tackles the underlying cause of CF in patients aged six to 11 with these mutations.

Symkevi has been an option for CF patients in this group aged 12 or over for two years, but the new approval means treatment will now be able to begin much earlier. As CF is a progressive disease, that’s an important consideration for patients and their parents.

The combination will be made available immediately to qualifying patients in Germany, according to Vertex, and will be available shortly in the UK and other countries that have reimbursement agreements with the drugmaker, including Denmark and Ireland.

“Today’s approval brings us closer to our ultimate goal of providing medicines for all people with CF,” said Vertex’s chief executive Reshma Kewalramani.

Vertex introduced Kalydeco in 2012, and has since followed up with launches of two-drug combinations Orkambi (lumacaftor/ivacaftor) and Symkevi – known as Symdeko in the US – in 2015 and 2018, respectively. Together these drugs cover the CFTR mutations seen in around half of all CF patients.

The next phase of Vertex is focused on the roll-out of its new triple Kaftrio (tezacaftor, ivacaftor and elexacaftor), which was launched last year in the US (as Trikafta) and was approved by the European Commission in August.

Kaftrio covers a broader set of CFTR mutations spanning 90% of CF patients, but for now is only available for the 12 and up age bracket, according to its EMA-approved label.

NHS England agreed an access deal for the new drug earlier this year, although Vertex has agreed to submit real-world data on Orkambi, Symkevi and Kaftrio to cost-effectiveness agency NICE which means the drugs’ price could be adjusted.

Sales of the Trikafta/Kaftrio approached $1 billion in the third quarter, driving a 62% increase in revenues at the company even as even as Vertex’ older therapies started to decline.

CF is a rare, life-shortening genetic disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas and reproductive tract. It is caused by a defective or missing CFTR gene, and there are around 75,000 people worldwide with the disease.

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Bio-Thera Reports MAA Submission to EMA for BAT1706 a Proposed Biosimilar to Avastin


  • The company has submitted an MAAA for BAT1706 to EMA. Bio-Thera seeks a commercial license for all approved indications of bevacizumab in the EU Member States, Iceland, Norway, and Liechtenstein
  • The submission of the MAA for BAT1706 marks it as the first ex-China MAA/ BLA submission. The BLA of the biosimilar for metastatic carcinoma of the colon or rectum and NSCLC is under NMPA’s review
  • The company plans to submit a BLA for BAT1706 to the US FDA by the end of 2020. Bevacizumab is a mAb that targets VEGF thus reduces neovascularization, thereby inhibiting tumor growth

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Businesswire

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Alvotech Reports the US FDA and EMA’s Acceptance of AVT02 Proposed Biosimilar to Humira (adalimumab)


  • The US FDA has accepted the BLA of AVT02 for review and is expected to decide on the filing in Sept’2021 while the EMA has accepted for review an MAA for AVT02 with an EMA decision anticipated in the Q4’21
  • The filings were based on AVT02-GL-101 & AVT02-GL-301 studies demonstrating a high degree of similarity b/w AVT02 and the reference products. AVT02-GL-101 study met its 1EPs of PK similarity while the later study confirmed the efficacy and safety of AVT02 in patients with mod. to sev. chronic psoriasis
  • AVT02 is a proposed biosimilar to the reference product Humira (adalimumab) with high concentration (100mg/mL) dosage forms

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: pharmaceutical daily

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Guido Rasi steps down, handing the running of EMA to Emer Cooke

EMA executive director Guido Rasi has ended his second term at the helm of the EU medicines regulator, with Emer Cooke taking the wheel and becoming the first women in the role.

Rasi’s second five-year term came to an end on Friday (13 November), with his second stint dominated by the move of the EMA offices from London to Amsterdam – and the associated staff losses and ongoing disruption that has resulted from the UK’s withdrawal from the EU – as well as the pandemic.

Cooke will take over the EMA as the regulator is in the midst of handling the review and authorisation of medicines for COVID-19, including the first vaccines for the coronavirus, and as the regulator continues to work out its future relationship with the UK Medicines and healthcare products Regulatory Agency (MHRA) – which has been a big contributor to its expertise and workload.

Under Rasi, the EMA implemented a suite of reforms including a drive towards transparency – for example making clinical study reports (CSRs) publicly available – and the implementation of sweeping EU directives on pharmacovigilance, clinical trials and falsified medicines.

During his tenure the EMA also implemented new rules to encourage the development of advanced therapy medicinal products (ATMPs) like gene and cell therapies, and the introduction of the PRIME fast-track review for promising medicines.

Emer Cooke

Cooke is returning to the EMA after a four-year period as head of medicines regulation at the World Health Organization (WHO). She first joined the EMA in 2002 and filled a number of senior roles at the regulator including head of inspections, head of international liaison and head of international affairs before switching to the WHO in 2016.

A pharmacist by training, Cooke has also worked in the pharma industry and at European industry group EFPIA, and according to Rasi is “a strong leader who is committed to steering EMA during this very challenging period and ensuring that the agency’s work on COVID-19-related activities continues uninterrupted.”

In his final message as head of the EMA, Rasi said the number of COVID-19 drugs and vaccines being developed by pharma companies is “encouraging,” and pointing to the agency’s pivotal role “in reviewing the available data packages to ensure that our usual high standards of safety, efficacy and quality are achieved and upheld.”

Cooke meanwhile told Bloomberg that one of the biggest challenges she will face immediately after re-joining the EMA will be monitoring the safety of COVID-19 vaccines, particularly those that are based on new technologies like mRNA.

She also told the newswire that the EMA is adopting a different stance on gauging the efficacy of COVID-19 vaccines from the FDA, which has placed a 50% reduction in disease severity as its threshold for approval, saying guidance on its position will be published soon.

The EMA has already started reviews of Pfizer/BioNTech’s mRNA-based shot BNT162b2 and AstraZeneca/University of Oxford’s adenoviral-based candidate AZD1222.

Rasi’s first five-year term as executive director of the EMA was interrupted in 2014 when his appointment was prematurely annulled by European Union Civil Service Tribunal – on the grounds that the selection process for the post was flawed – only to resume his duties a year later.

Prior to the EMA role, Rasi was director-general of the Italian Medicines Agency from 2008, and previously spent three years as director of the Institute of Molecular Medicine in Rome.

“I am leaving EMA at a time when its mandate is set to be expanded. This is an acknowledgement of the good work and reputation that the agency has built over the last 25 years,” he said.

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Bayer Reports Regulatory Submission of Finerenone (BAY 94-8862) to the US and EU for CKD and T2D


  • The MAA submitted to the EMA and the NDA to the US is based on P-III FIDELIO-DKD study assessing finerenone (10/20mg, qd) + SOC vs PBO in ~5,700 patients with CKD and T2D
  • Result: reduction in the combined risk of time to kidney failure; a decrease of eGFR (≥ 40%) from baseline @4wks. or renal death by 18%, reduction in risk of 2EPs i.e. time to CV death, non-fatal MI, non-fatal stroke, or hospitalization for HF by 14% over a median duration of follow-up of 2.6yrs.
  • Finerenone is a selective MRA, being evaluated in FIGARO-DKD study in ~7,400 patients with CKD and T2D. Bayer has also initiated P-III FINEARTS-HF study of finerenone in symptomatic HF patients with a left ventricular ejection fraction of ≥40%

Click here ­to­ read full press release/ article | Ref: Bayer | Image: The Business Journals

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Trials and tribulations in Biogen’s Alzheimer’s drug reviews

Regulatory reviews of Biogen’s Alzheimer’s drug aducanumab are now ongoing on both sides of the Atlantic, but debate is still ongoing about whether the data behind the drug is strong enough to support approval.

The EMA has just kicked off its review of the anti-amyloid therapy, following in the footsteps of the FDA in the US which has been looking at the drug since August, but a new analysis of the mixed phase 3 data for aducanumab argues that an additional trial should be carried out.

The paper in the journal Alzheimer’s & Dementia, led by Mayo Clinic neurologist David Knopman, says that efficacy of aducanumab “as a treatment for the cognitive dysfunction in Alzheimer’s disease cannot be proven by clinical trials with divergent outcomes.”

Meanwhile, the paper also notes that Knopman has been excluded from an FDA advisory committee meeting due to discuss the data on Friday, ahead of a decision on the marketing application due in March.

The expert – who was an investigator in the phase 3 trials of Biogen’s drug – told Reuters he was recused from the panel because of his involvement in conducting clinical trials of aducanumab.

Aducanumab – which Biogen is developing with Japanese drugmaker Eisai – was all but abandoned in 2019 after the partners decided that two phase 3 trials of the drug were unlikely to show an effect on cognitive decline in Alzheimer’s.

Shares in Biogen were hit hard, as investors lost hope that aducanumab might be rescue the almost defunct amyloid hypothesis of Alzheimer’s disease, which holds that blocking the formation of amyloid plaques in the brain could delay the onset of dementia.

Just a few months later however they said a fresh look at the results of the EMERGE and ENGAGE studies had revealed that the initial futility analysis was “incorrect.” In fact, the drug reduced clinical decline in patients with early, a chance was put down to more exposure to a higher dose in additional patient follow-up.

Some patients showed statistically significant improvements on symptoms like memory, orientation, and language, as well as being able to carry out day-to-day tasks more easily.

There’s a lot riding on the FDA and EMA reviews. If approved, aducanumab will become the first therapy to reduce the clinical decline of Alzheimer’s and to change the course of the disease, says Biogen. It would also be the first amyloid-targeting drug to reach the market, after dozens of others have failed in clinical development.

Meanwhile, aducanumab is the big hope in Biogen’s late-stage pipeline, which otherwise is looking fairly thin, at a time when the biotech is facing the loss of patent protection for its blockbuster multiple sclerosis therapy Tecfidera (dimethyl fumarate).

Knopman and fellow authors argue in the Alzheimer’s & Dementia paper that Biogen’s interpretation of data in the two trials might not be correct.

They write that they have found alternative explanations for the apparent drug benefits unrelated to the treatment, and say that while there is evidence that aducanumab was working on amyloid and other biomarkers like tau protein as expected, “no evidence was presented to correlate biomarker changes to cognitive benefits.”

They also say there were differences in the placebo responses between the two studies, which could have contributed to the divergent results.

“Our analysis supports the conduct of a third, definitive phase 3 trial with high‐dose aducanumab [that is] optimally designed and adequately powered to prove efficacy,” they conclude.

The FDA has not commented on the reasons for Knopman’s exclusion from the advisory committee meeting publicly, but in these cases there is usually a conflict of interest.

Along with his involvement in EMERGE and ENGAGE, Knopman also serves on a data safety monitoring board for a tau drug for Alzheimer’s developed by Biogen, and is an investigator in a trial sponsored by Eli Lilly and the University of Southern California.

He also performs unpaid consultancy work for Samus Therapeutics, Third Rock, Roche, and Alzeca Biosciences, according to the paper’s conflict of interest statement.

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FDA sets April verdict for Pfizer’s “underestimated” atopic dermatitis drug

Pfizer could be just a few months away from getting FDA approval for its JAK1 inhibitor abrocitinib in atopic dermatitis, a drug that CEO Albert Bourla believes hasn’t been given the credit it is due by Wall Street analysts.

The US regulator has started a priority review of abrocitinib for mediate to severe atopic dermatitis in patients aged over 12, setting up a decision next April, while the EMA has also started a standard review that could lead to approval in the EU in the second half of 2021.

Bourla told analysts on Pfizer’s results call yesterday that abrocitinib is “the one potential near-term compound where we see the biggest difference compared with consensus.”

Pfizer sees abrocitinib and other JAK drugs coming through the pipeline for atopic dermatitis as expanding the number of patients getting treatment for the more severe end of the spectrum of symptoms – and it reckons that unlocks blockbuster sales potential.

“This is not a zero-sum game with the biologics in the treatment of moderate to severe atopic dermatitis,” said Bourla.

These patients currently rely on biologic drugs like Sanofi and Regeneron’s Dupixent (dupilumab), which dominates the category and saw sales rocket to more than $2 billion last year.

Sanofi sees plenty of additional upside, suggesting the drug could become a $10 billion brand at peak from expansion in atopic dermatitis as well as new indications like asthma, chronic rhinosinusitis with nasal polyps and eosinophilic oesophagitis.

Abrocitinib has previously been shown as effective in the JADE-MONO-1 and JADE-MONO-2 trials in subjects aged over 12, with a profile that looks like it could challenge its biologic rivals.

In particular, Pfizer says the drugs seem to have an impact on itch – often cited as the most bothersome symptom by atopic dermatitis patients.

Bourla and Angela Hwang – group president of Pfizer’s biopharmaceuticals division – think analysts are overlooking the sheer scale of atopic dermatitis as a condition with around 60 million sufferers aged over 12 worldwide.

They are also discounting abrocitinib’s profile, and the fact that almost two-thirds of patients treated with Dupixent don’t achieve clear or almost clear skin at 16 weeks, leaving room for improvement.

“Of those 60 million, only 7% of them today are being treated with a systemic agent,” said Hwang on the call. “So the systemic market opportunity has real potential to more than double with the introduction of better systemic treatment, because the patient need is just so high.”

There’s a precedent for that level impact for new biologic drugs such as interleukin inhibitors for psoriasis, which doubled the market over a 10-year period.

Claiming just 8% of the systemic atopic dermatitis treatment market would equate to $3 billion in abrocitinib sales, said Hwang.

Pfizer’s drug seems to have a lead in atopic dermatitis over other JAK inhibitors, although AbbVie has trials on the go for its fast-growing Rinvoq (upadacitinib) rival in this indication – including a head-to-head comparison with Dupixent due to read out next year.

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Kite cues up first EMA okay for mantle cell lymphoma CAR-T

Gilead Sciences’ Kite Pharma unit is closing on approval of its second European approval for a CAR-T for cancer, after the CHMP backed its Tecartus therapy for mantle cell lymphoma.

The EMA’s human drugs advisory committee recommended approval of Tecartus (brexucabtagene autoleucel; formerly KTE-X19) for relapsed or refractory MCL setting up a formal approval by the regulator in the coming weeks.

Tecartus is the third CAR-T therapy to be recommended for approval in Europe and will be the first for MCL, an aggressive form of non-Hodgkin’s lymphoma (NHL).

MCL is generally treated first with chemotherapy, and patients whose disease progresses despite that can then receive stem cell transplantation or BTK inhibitor drugs like Johnson & Johnson/AbbVie’s Imbruvica (ibrutinib) and AstraZeneca’s Calquence (acalabrutinib).

Despite these therapies, relapsed/refractory MCL patients often relapse or stop responding to treatment, according to the EMA.

Tecartus was cleared by the US FDA in the summer, and was the first CAR-T from Kite to launch without direct competition, as its first therapy Yescarta (axicabtagene ciloleucel) is going head-to-head in the market with Novartis’ rival Kymriah (tisagenlecleucel).

Gilead will be hoping that Tecartus will inject some additional momentum into its CAR-T franchise, which underpinned its $11.9 billion acquisition of Kite in 2017.

So far, Yescarta – which is approved for other forms of NHL – has performed well below its blockbuster sales projection when it launched three years ago, bringing in $296 million in the first half of this year. Kymriah hasn’t delivered as hoped for Novartis either, which has been attributed to manufacturing problems.

Yescarta’s lacklustre performance caused Gilead to take an $800 million charge in the fourth quarter of 2019, and that came after an $820 million write-down a year earlier as the company downgraded the value of Yescarta and Kite’s CAR-T pipeline.

Tecartus has been recommended for approval in the EU on the strength of the ZUMA-2 trial, which involved 74 adult MCL patients who had previously been treated with at least two prior therapies, including BTK drugs.

Over 12 months of follow-up, 84% of patients treated with Tecartus responded to treatment, with 59% showing a complete response, a level of efficacy that goes further even than the impressive results achieved by Yescarta.

Tecartus employs the same T-cell construct as the previously approved CAR-T Yescarta but has a slightly modified manufacturing process to boost its activity in diseases where there is a large burden of circulating tumour cells.

The EMA designated Tecartus a Priority Medicine (PRIME), allowing for an expedited review.

The CAR-T is also being tested in acute lymphocytic lymphoma (ALL), and if approved that will place the CAR-T in competition with Kymriah, which has been approved to treat ALL since 2017.

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Novartis’ big bet on inclisiran nears fruition, as CHMP backs drug

Novartis’ near-$10 billion takeover of The Medicines Company last year was focused mainly on one asset – cholesterol-lowering drug inclisiran – and the Swiss pharma is now a step closer to getting a return on its investment.

Inclisiran – now give the trade name Leqvio – has been recommended for approval by the CHMP as a treatment for hypercholesterolaemia or mixed dyslipidaemia, two common forms of elevated cholesterol linked to increased risk of cardiovascular disease.

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients.

The drug inhibits PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Novartis is hoping that with Leqvio it will make a mark where the antibody drugs have largely failed, with sales of Repatha and Praluent still failing to gather much momentum despite being on the market for more than five years, after pushback from payers that wasn’t overcome by substantial price cuts.

The big question for Novartis is whether Leqvio’s more convenient dosing will be enough for it to overtake its antibody-based rivals, given that they seem to be fairly equivalent when it comes to lowering LDL-C.

Novartis’ drug is also playing catch-up with its rivals on data from trials that show the reduction in LDL-C is matched by an improvement in cardiovascular outcomes.

Both Repatha and Praluent have that in hand, but Novartis will have to wait for the results of the ORION-4 study – due in 2024 – before it has a chance of matching the labels of Amgen and Sanofi/Regeneron’s drugs.

That said, Novartis is no stranger to building a slow-starting cardiovascular drug into a blockbuster franchise. It’s a trick it carried out with Entresto (sacubitril/valsartan) – a drug for heart failure – that after a sluggish roll-out is now bringing in sales at a rate of more than $2 billion a year.

That also means the Swiss drugmaker will also be able to push Leqvio through the sales channels that have made Entresto a blockbuster, but it will need to make swift progress if it is to get a reasonable return on the investment in MedCo.

Evercore ISI analyst Umer Raffat has previously said he thinks that will come if Leqvio hits $2 billion in peak annual sales.

Novartis already has some deals in place that could help it reach that target, including an access pact with NHS England aimed at accelerating access to Leqvio after EMA approval. Billed by Novartis as a “world-first”, the deal would see the drug made available to people with atherosclerotic cardiovascular disease.

Earlier this year analysts at Clarivate said they expect Leqvio to reach $1.16 billion in sales by 2024 as a treatment for elevated blood cholesterol among the 80% or so of patients who don’t respond to older statin drugs.

The CHMP sets up a likely EMA approval for Leqvio before the end of the year, and Novartis is also expecting to hear back from the FDA on the drug in the same timeframe.

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BioNTech and Pfizer Initiates Rolling Submission to EMA for SARS-CoV-2 Vaccine Candidate, BNT162b2


  • The Rolling Submission is based on the preliminary results from pre-clinical and early clinical studies in adults, which shows that BNT162b2 triggers the production of neutralizing antibodies and TH-1 dominant CD4+ and CD8+ T cells that target SARS-CoV-2. BioNTech and Pfizer plan to work with the EMA’s CHMP to complete the rolling review process to facilitate the final MAA
  • BNT162b2 vaccinated participants showed a favorable breadth of epitopes recognized in T-cell responses specific to SARS-CoV-2 spike antigen & BNT162b2 demonstrated concurrent induction of high magnitude CD4+ & CD8+ T cell responses which are TH-1 dominant against RBD and remainder of full spike glycoprotein
  • BNT162b2 vaccine candidate (BioNTech’s proprietary mRNA technology and supported by Pfizer) encodes an optimized SARS-CoV-2 full-length spike glycoprotein (S). It is currently being evaluated in an ongoing P-III study with ~37,000 participants enrolled and 28,000 having received their second vaccination at >120 clinical sites worldwide including the US, Brazil, South Africa, and Argentina

Click here ­to­ read full press release/ article | Ref: GlobeNewswire | Image: King’s College London

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Race for COVID-19 vaccine hots up as EMA begins review of BioNTech/Pfizer jab

The European Medicines Agency has begun a second “rolling review” of a potential coronavirus vaccine jointly developed by BioNTech and Pfizer, setting up a race with a rival from AstraZeneca and Oxford University.

The announcement comes just days after the regulator said its CHMP scientific committee had begun to look at the first batch of data from AZ’s rival.

In the case of a rolling review, the EMA evaluates trial data as they become available before a complete filing is submitted.

It’s hoped that this will hasten the process, which would take more than a year in normal conditions.

Once the CHMP thinks there is enough data the company submits the complete filing, allowing the committee to reach a fast decision, which is passed on to the European Commission.

The CHMP begun its review of the vaccine, known as BNT162b2 based on preliminary results from pre-clinical and early clinical studies.

These studies suggest BNT162b2 triggers the production of neutralising antibodies and T-cells that target SARS-CoV-2, the coronavirus that causes COVID-19.

The vaccine is based on different technology from AZ’s rival: while AZ’s uses a weakened and genetically modified cold virus to prime the immune system, BNT162b2 is based on a string of RNA.

The RNA instructs the body to produce copies of the Spike protein found on the surface of the coronavirus, which produces an immune response and causes the body to neutralise the virus if it infects the body.

BNT162b2 is being tested in a global phase 3 study ongoing at more than 120 clinical sites worldwide including the US, Brazil, South Africa and Argentina.

To date, the trial has enrolled approximately 37,000 participants with more than 28,000 having received their second vaccination.

According to latest figures from the World Health Organization, there are 42 potential COVID-19 vaccines in clinical development.

It lists a candidate developed by China’s Sinovac as the most advanced, with AZ’s shot now fourth on the list after trials were temporarily halted because of a potential safety issue.

Pfizer’s is further back on the list, although there are suggestions that it could be approved in the US in time for the presidential elections on 3rd November.

US trials of AZ’s vaccine have not restarted while the FDA investigates the safety issue that caused the trials to be paused around a month ago.

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EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years’ follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donor’s immune cells attack the recipient’s cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patient’s own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

“CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death,” said Gary Fortin, head of severe genetic disease programmes at Bluebird.

“If approved, eli-cel would represent the first therapy for CALD that uses a patient’s own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor,” he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebird’s development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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Galecto’s GB0139 Receives the US FDA’s and EMA’s Orphan Drug Designations for Idiopathic Pulmonary Fibrosis


  • The US FDA and EMA has received ODD to Galecto’s GB0139 for the treatment of IPF. GB0139 showed significant reduction of YKL-40 biomarker in fibrosis, inflammation, tissue remodeling diseases in its first clinical study after 14 days of treatment
  • The EMA cited GB0139’s clinically relevant biomarker data in IPF patients which provides financial incentives, encouraging the development of drugs targeting rare diseases
  • GB0139 (formerly TD139) is an inhaled galectin-3 inhibitor, being evaluated in P-IIb GALACTIC-1 study in 450 patients with IP across 100 centers in the US the EU and Canada

Click here, ­to­ read full press release/ article | Ref: PRNewswire | Image: Galecto

PTC Reports the EMA’s Acceptance of MAA for Evrysdi (risdiplam) to Treat SMA


  • The MAA submission is based on dose-finding Part 1 and confirmatory Part 2 of the FIREFISH and SUNFISH studies evaluating the efficacy and safety of Evrysdi (risdiplam) in symptomatic infants with type 1 SMA aged 2-7mos. and in people with types 2/3 SMA aged 2-25 yrs/ respectively
  • The submission also includes data from JEWELFISH study evaluating patients with all types of SMA aged 1-60yrs. prior treated with other SMA therapies. Exploratory efficacy analysis from SUNFISH Part 1 showed Evrysdi improved motor function @24mos. of treatment, Part 2 of the FIREFISH study met its 1EPs of infants sitting without support for 5sec by month 12, as assessed by BSID-III, Part 2 of the SUNFISH trial demonstrated that change from baseline in MFM-32 score was greater at 12mos.
  • Roche will grant $15M as milestones to PTC following MAA acceptance. Evrysdi is an SMN2-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q leading to SMN protein deficiency

Click here to­ read full press release/ article | Ref: PRNewswire | Image: PTC Therapeutics

Insights+ Key Biosimilars Events of July 2020

Biosimilars are developed to be highly similar versions of approved biologics in terms of safety, purity, and potency. Biosimilars are expected to be a cost-effective alternative to the high-priced branded biologics, offering significant and much-needed cost savings to both payers and the patients. Hence, the providers are more likely to adopt biosimilars as a “reference product to biologics” possessing similar therapeutic properties. Mylan with its partner Kyowa Kirin received the US FDA’s approval for its adalimumab biosimilar, Hulio (adalimumab-fkjp). Cadila expanded its footprints in India with the launch of its two biosimilars Bevaro (bevacizumab, biosimilar) and Ritucad (rituximab, biosimilar). Our team at PharmaShots has summarized 9 key events of the biosimilar space of July 2020.

1. Samsung Bioepis Reported the Initiation of P-III Study for SB15 Proposed Biosimilar to Eylea (aflibercept)

Date: Jul 01, 2020  

Product: SB15 Biosimilar

  • The P-III study compared the efficacy, safety, PK, and immunogenicity between SB15 and Eylea in 446 patients with neovascular age-related macular degeneration
  • The company has two ophthalmology biosimilar candidates in clinical development, SB11 (ranibizumab) and SB15 (aflibercept). On May 18, 2020, Samsung Bioepis reported 24-week interim results from a P-III study of SB11 (proposed ranibizumab biosimilar)
  • SB15 is a proposed biosimilar referencing Regeneron’s Eylea, currently under preclinical treatment for diabetic macular edema, diabetic retinopathy, retinal edema, and retinal vein occlusion

2. Mylan and Fujifilm Kyowa Kirin Received the US FDA’s Approval for its Hulio (biosimilar, adalimumab)

Date: Jul 09, 2020

Product: Hulio (adalimumab-fkjp)

  • The US FDA has approved Hulio (adalimumab-fkjp), a biosimilar to AbbVie’s Humira (adalimumab) to treat RA, JIA (in patients aged ≥4yrs.), PsA, AS, adult CD,  UC, and PsO, in both prefilled syringe and auto-injector presentations
  • The approval was based on an analytical, pre/ clinical program. The P-III ARABESC study conducted by Fujifilm Kyowa Kirin, demonstrated no differences in terms of safety, efficacy, and immunogenicity compared to its reference product in patients with RA
  • In 2018, the companies collaborated to commercialize Hulio in the EU while expanded the agreement globally in 2019. As per the patent license agreement with AbbVie, Mylan will be able to launch Hulio in the US in Jul’2023

3. Bio-Thera Signed a License Agreement with Pharmapark for BAT2506 (golimumab, biosimilar)

Date: Jul 17, 2020  

Product: BAT2506 (golimumab, biosimilar)

  • Bio-Thera will take care of full development, and commercial supply of BAT2506 out of the manufacturing facilities in Guangzhou, China. Additionally, the agreement will utilize Pharmapark’s local presence, sales, and marketing capabilities in Russia and other CIS countries
  • Pharmapark to get exclusive rights to distribute and market the product in Russia and other CIS countries including its filing practices. In Q4’20, Bio-Thera to begin its global P-III trial in PsA patients and will include patients from China, Russia, and many other countries 
  • Bio-Thera’s BAT2506 is a mAb while the company plans for regulatory submission with NMPA, EMA and the US FDA in 2023

4. Cadila to Launch its Bevaro (bevacizumab, biosimilar) in India

Date: Jul 22, 2020  

Product: Bevaro (bevacizumab, biosimilar)

  • Cadila plans to launch multiple biosimilars including Bevaro in 2020 which are safe and efficacious compared to conventional treatment therapies
  • The company has also cleared he USFDA inspection in Feb’20 and holds approved 16 ANDAs plus currently filed 30 ANDAs  
  • Bevaro is a mAb will be available in two strengths of 100 and 400 mg targeted for Ovarian Cancer, Glioblastoma Multiforme (a type of Brain cancer), Colorectal Cancer, Breast Cancer, Lung Cancer, Cervical Cancer and Kidney Cancer in India

5. Centus Biotherapeutics’ Equidacent (bevacizumab, biosimilar) Received CHMP’s Positive Opinion for the Treatment of Multiple Cancer Indications

Date: Jul 27, 2020

Product: Equidacent (bevacizumab, biosimilar)

  • The EMA’s CHMP has adopted a positive opinion that recommended the MAA of Centus’ Equidacent (bevacizumab), a biosimilar to Roche’s Avastin to treat carcinoma of the colon or rectum, breast cancer, NSCLC, RCC, epithelial ovarian, fallopian tube or primary peritoneal cancer, and carcinoma of the cervix
  • Equidacent will be available as 25 mg/ml as the solution for infusion targeting VEGF and act by inhibiting the binding of VEGF to its receptors on endothelial cell
  • The company anticipates EC’s decision in ~60days. The clinical data demonstrated that Equidacent has comparable quality, safety, and efficacy to Avastin

6. NCCN Collaborated with Pfizer to Explore Adoption of Biosimilars in Oncology

Date: Jul 27, 2020

Products: Biosimilars

  • NCCN and Pfizer issued an open RFP that can develop and validate enduring approaches to improve the safe, effective, and efficient adoption of biosimilars in oncology
  • The RFP supports approaches to improve the processes related to biosimilar adoption in oncology. Letters of Intent are welcome from academic or community centres, patient advocacy groups, and HCPs organizations in the US and are due on Sept 09, 2020
  • The NCCN ORP will boost up the discovery that improves the lives of people with cancer and supports preclinical, translational, clinical research and quality improvement projects in oncology

7. Celltrion’s Remsima SC (infliximab, CT-P13) Received the EC’s MAA for Additional Five Indications

Date: Jul 27, 2020  

Product: Remsima SC (infliximab, CT-P13)

  • The approval followed the EMA’s CHMP positive opinion based on a study assessing Remsima SC (120 mg) vs IV formulations in patients with Crohn’s disease and ulcerative colitis, evaluating its PK, efficacy, and safety
  • The study resulted in high efficacy and safety comparable with IV formulation and the SC administration will also reduce the treatment time to approximately 2-5 minutes
  • Remsima is a world’s first mAb indicated to treat eight autoimmune diseases including RA & IBD and approved in the US & EU with Inflectra & Remsima respectively. Till now the product is approved in 94 countries including the US, Canada, Japan and throughout EU

8. Cadila to Launch its Ritucad (rituximab, biosimilar) in India

Date: Jul 27, 2020  

Product: Ritucad (rituximab, biosimilar)

  • Cadila to strengthen its biosimilar portfolio and oncology footprints in India with the launch of Ritucad and has cleared USFDA inspection in Feb’20
  • Additionally, the company has launched its Bevaro (bevasizumab, biosimilar) a mAb targeted for multiple cancer indications like Ovarian Cancer, Glioblastoma Multiforme, Colorectal Cancer, Breast Cancer, Lung Cancer, Cervical Cancer and Kidney Cancer in India
  • Ritucad is the second biosimilar in Cadila’s portfolio of biosimilars and will be available in a single-dose vial of two strengths, 100 mg & 500mg targeted to treat Non-Hodgkin’s lymphoma and RA

9. Henlius and Accord’s Zercepac (trastuzumab, biosimilar) Received the EMA Approval for HER2-Positive Breast Cancer and Gastric Cancer

Date: Jul 30, 2020

Product: Zercepac (trastuzumab, biosimilar)

  • The approval was based on comparative quality studies, clinical studies including preclinical data and resulted in bio-similarity, comparable efficacy, and safety of Zercepac (HLX02) vs Herceptin
  • Zercepac is developed under NMPA and EMA biosimilar guidelines and has been evaluated with the reference trastuzumab including comparative quality studies, preclinical studies, a P-I clinical study and a global multi-centre P-III clinical study
  • Zercepac is a novel China-mAb approved in the EU for the treatment of HER2+ early breast cancer, HER2+ metastatic breast cancer, and HER2+ metastatic gastric cancer with an expected launch in China in 2020 while its manufacturing site has received EU GMP certificates

Related Post: Insights+ Key Biosimilars Events of June 2020

UCB’s Cimzia (certolizumab pegol) Receives the EMA’s Approval for a Reduced Maintenance Dose in Patients with Axial Spondyloarthritis Spectrum


  • The P-IIIb C-OPTIMISE study assessing Cimzia (200mg, q2w with a loading dose of 400mg @ 0, 2 & 4wks.) vs PBO during 48wks. open-label induction period in adults with early active axSpA. At 48wks., patients in sustained remission (ASDAS <1.3 @wks. 32/36 & 48) were randomized to Cimzia 200mg, q2w (full maintenance dose) & 200mg q4w (reduced maintenance dose) or PBO (withdrawal) for an additional 48wks.
  • The EMA label extension is based on the results of the C-OPTIMISE study that demonstrated @48wks. 43.9% of patients achieved sustained remission, @96wks. 84%, 79% & 20% of patients receiving the full maintenance dose, reduced maintenance dose or PBO respectively remained flare-free
  • The approval makes Cimzia the only biologic in EU with a dose reduction option in its label for patients in the broad axSpA population

Click here ­to­ read full press release/ article | Ref: UCB | Image: CHE Manager

Sanofi faces possible manslaughter charges in epilepsy drug probe

Sanofi has been placed under formal investigation in France for possible charges including manslaughter relating to the epilepsy drug Depakine, following the deaths of four babies whose mothers took the drug.

Depakine was found to carry a high risk of birth defects if taken by pregnant women.

Sanofi, which denies wrongdoing and says it warned of the risks long ago, is already being prosecuted in France following allegations it provided misleading information about the drug.

The drug is widely prescribed under different names as a treatment for epilepsy and other forms of seizure.

Although there have been warnings for years that the drug should not be given to pregnant women, complainants in France say this was never adequately communicated.

As a result thousands of children were born with physical or psychological problems because of the increased risk of birth defects or autism in their babies.

It is unclear whether the matter will go to trial, the BBC reported.

A spokesperson for Sanofi said: “As part of the investigation on Depakine opened at the Court of Justice of Paris for nearly four years, Sanofi Aventis France was indicted for aggravated deception, involuntary injuries and recently for manslaughter.

“The pharma company has referred the matter to the Chamber of Inquiry in order to challenge that indictment.

“All these elements prejudice in no way the responsibility of the pharma company.

“Sanofi Aventis France has complied with its disclosure obligations and is challenging the merits of these lawsuits.”

In 2018, the European Medicines Agency’s safety committee recommended new measures to avoid unborn babies becoming exposed to valproate.

The Pharmacovigilance Risk Assessment Committee (PRAC) made recommendations including a visual warning about the risks in pregnancy, possibly including a symbol or pictogram to be adapted at national level.

In migraine or bipolar disorder the PRAC said valproate must not be used during pregnancy, and not in women who are thinking of having babies.

In epilepsy the PRAC said it must not be used unless it is impossible to discontinue treatment.

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BioMarin Reports the Submission of MAA to EMA for Vosoritide to Treat Children with Achondroplasia


  • The MAA is based on P-III study assessing the efficacy and safety of vosoritide, further supported by the long-term safety and efficacy from the ongoing P-II and P-III extension studies and extensive natural history data
  • Following the completion of EMA’s validation, BioMarin expects the initiation of the MAA review to commence in Aug’2020. The company is planning to file NDA to the US FDA in Q3’20
  • Vosoritide is an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia and has received FDA’s and EMA’s ODD

Click here to read full press release/ article | Ref: BioMarin | Image: BioMarin

How Are Regulatory Agencies Reacting to the Use of Real-World Evidence?

As we have discussed here previously, real-world data (RWD) and real-world evidence (RWE) offer many potential benefits in every stage of the drug discovery and development process, continuing on into post-market surveillance. With drug developers and other researchers becoming more interested in using RWD and the RWE that results from analyzing it, regulatory agencies have had to step up and work on producing guidance.

There are many
challenges that accompany RWD. Its various forms (e.g. EHRs, disease
registries, claims data) are not necessarily subject to the same
well-established regulations and protocols as clinical data. The data might be
inconsistent, unstructured, in multiple formats and it may not adhere to the
principles of FAIR data. As regulatory bodies consider RWE, they must think
about the quality of the data underpinning it.

The FDA offers

The Food and Drug Administration (FDA) took its first big step in December 2018 by publishing a framework for their real-world evidence program, which helped to lay out some of their goals and issues of importance to be addressed, such as how RWE will be used for regulatory decision-making for drugs, considerations for observational study designs and clinical trial design, data standards for submissions, regulatory issues around the use of electronic source data and more. Actual draft guidance for submitting documents using RWD and RWE for drugs and biologics then followed in May 2019.

The EMA grapples with real-world

Meanwhile in Europe, the European Medicines Agency (EMA) has also had to address the intense interest in RWD and RWE, though there are clearly concerns about whether real-world evidence can be credible evidence. In an article published in the journal Clinical Pharmacology & Therapeutics in October 2019, the EMA officials who authored it noted concerns that “acceptance of non‐RCT methodologies is tantamount to lowering the quality of evidence because these methods are prone to a myriad of undetected or undetectable biases.”

They remain optimistic
about the future for RWE, but are adamant about the importance of testing and
validation. “The ultimate key to achieving credibility is to start with an open
but ‘agnostic’ mind‐set and submit novel
methods to a fair, transparent, and prospective validation exercise,” they wrote.

The pharma response

The FDA has invited comments on its draft guidance, and the pharmaceutical industry has obliged. As reported in Policy & Medicine, a number of suggestions have come in from major players. Gilead, for instance, has proposed expanding the submissions list so that supplemental new drug applications and supplemental biologics license applications are included. Gilead has also suggested lab data be considered a source of RWD, and Novartis has suggested pharmacy claims should be considered a source for RWE. 

What is quite clear is
that we are in the early stages of what will be a long process, as regulators
work to formulate policy and guidance for a type of data that they are still
trying to fully define. Real-world data and real-world evidence have much to
offer in drug development and post-market, and it will be important to have the
guidance and cooperation of our most influential regulatory bodies.

In our next piece on
RWE, we will discuss the role of real-world evidence in the fight against
COVID-19, including a new research project spearheaded by the FDA.