FDA
Last month, the FDA committed to designing a “fit-for-purpose” framework for regulating rare disease therapies based on patient need and realistically attainable evidence. With the upcoming decision for Capricor’s Duchenne muscular dystrophy cell therapy deramiocel, this paradigm faces its first key test.
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Inclusion of the patient perspective is currently an “optional” part of the rare disease drug development process, CBER Director Karim Mikhail told BioSpace. Mikhail, along with his CDER counterpart Michael Davis and other agency rare disease leaders, is trying to change that.
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
By allowing compounding pharmacies to produce certain unapproved peptides, the Department of Health and Human Services could, in the short term, put patients in harm’s way. Longer term, the policy shift threatens the industry that brings new medicines to market.
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After Novartis confirmed the reports of tumors in patients who have been treated with the spinal muscular atrophy gene therapy, the FDA will determine if any regulatory action is needed for Zolgensma or for the pharma’s similar product Itvisma.
After the FDA delayed a decision for Agios Pharmaceuticals’ sickle cell disease candidate, the biopharma is letting go of more than 35% of its R&D employees.
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is designed to develop personalized gene therapies more quickly.
If approved, BridgeBio Pharma’s BBP-418—which, according to one Jefferies analyst, boasts “pretty darn compelling” data—would be the first therapy for limb-girdle muscular dystrophy. The FDA has an action date of Nov. 27.
Under the interim policy, officials do not intend to take action against sites that produce the peptides.
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
Drug developers increasingly have the option of seeking regulatory approval and commercializing a medicine themselves, rather than accepting an early exit offer. Doing so requires a worldwide strategy.
After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aardvark plans to resume testing the candidate next year.