Rare diseases–also go by the name Orphan diseases–are the conditions that do not affect many individuals out there in one particular geographic area. Going deeper into the definition, it also means a lesser patient pool for a therapy developed for one identified rare condition. Unfavorable… Read More »What are the Market Drivers Fueling the Rare Disease Market?
Rare diseases affect a fraction of the population – 450 million+ people globally. However, its effects can be equally severe and life-threatening as other prevalent diseases. For Rare Disease Day, Tarun Kumar Bansal discusses why we need to expedite innovation in the rare disease domain.… Read More »Expediting innovation in the rare disease domain
Chiesi and Protalix have built the case for their long-acting enzyme replacement therapy (ERT) for Fabry disease PRX-102, as the FDA’s review of the drug continues towards a decision due by 27 April. New data from the phase 3 BRIGHT study of PRX-102 (pegunigalsidase alfa)… Read More »Switch data back Chiesi/Protalix long-acting Fabry drug
Lack of knowledge about conditions, situations, or body ailments that are unexplainable often scares people and leads to people jumping to conclusions or say myths that are not at all backed by science and logic. Myths are a seemingly convenient way to explain what the… Read More »Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook
Like troops at the forefront of any battle, our immune system continually marches to protect us from foreign elements and invaders, including bacteria, viruses, and others. Whenever with a swift wave of wind, something falls in our eyes, our immune system produces tears to get… Read More »Hypofibrinogenemia Market: Limited Pipeline Therapies, Abundant Opportunities
Ensoma contends its technology avoids the complexities of other approaches to gene therapy, which could expand genomic medicine’s reach to more patients and more diseases. Takeda Pharmaceutical is collaborating with the startup on up to five gene therapies.
How is a Rare disease defined? Well, it depends on the geography in question, since there is no universal definition, even so, the definition revolves around the point of prevalence or incidence of rare disease. However, despite the changing attributes in the definition, there is… Read More »The Paradox of Rare: Chalking out Challenges in the Rare Disease Market
Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter.
The FDA approval clears Regeneron’s drug, Evkeeza, as a treatment for the rarer of two forms of an inherited disease that causes high cholesterol levels. The company already markets Praluent, which treats the more common form of familial cholesterolemia.
One year after losing Merck as a partner, KalVista reports mid-stage clinical data for a pill that could offer an alternative to injectable drugs for a rare, potentially life-threatening disease. KalVista plans to meet with the FDA about proceeding to a Phase 3 study.
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… Read More »Sobi gets EU nod for Doptelet in rare blood disorder ITP
A survey by rare disease patient network Raremark found that 86% of the community members asked were interested in taking part in clinical trials. CEO Jeremy Edwards looks at how decentralised trial models can solve some of the challenges for clinical trial recruitment in rare… Read More »Unlocking the potential in rare disease research with decentralised trials
Johnson & Johnson’s Darzalex Faspro has become the first product approved by the FDA to treat light chain (AL) amyloidosis, a rare and often fatal blood cell disorder. The new indication is the second for Darzalex Faspro, a subcutaneous version of blockbuster intravenous therapy Darzalex… Read More »J&J’s Darzalex Faspro is first US therapy for rare blood disorder
To help clinicians diagnose rare disease more quickly and accurately, many healthcare organizations are embracing technology solutions like natural language processing (NLP) tools that can create augmented intelligence workflows that facilitate the rapid search of unstructured clinical data from multiple data sources.
How do you incentivize pharmaceutical companies and researchers to develop new drugs for rare diseases? Drug development is expensive and–by definition–rare diseases have a small market. One solution to this problem is that governments have used orphan drug policies to provide monetary incentives for innovators… Read More »Orphan Drug Policies Around the World
Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. Rare disease specialist Sarepta is putting up $15 million of that first-round financing,… Read More »Sarepta helps bankroll new gene therapy startup AavantiBio
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… Read More »EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD
The FDA has started a speedy review of Regeneron’s ANGPTL3-targeting antibody evinacumab for a rare, inherited disorder that dramatically raises the risk of heart disease. The cholesterol-lowering drug has been classed as a breakthrough therapy for homozygous familial hypercholesterolaemia (HoFH) by the FDA, which is… Read More »Regeneron’s cholesterol drug evinacumab claims February FDA date
The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by… Read More »Chiesi’s flagship Fabry drug heads for FDA verdict in early 2021
An FDA advisory committee has narrowly voted in favour of approval of Mallinckrodt’s terlipressin for hepatorenal syndrome type 1 (HRS-1), after the agency questioned the findings of a phase 3 trial. The US regulator doesn’t have to approve on the strength of the 8 to… Read More »Mallinckrodt’s liver disease drug terlipressin edges FDA advisory panel