Regulatory
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.
Beam Therapeutics’ risto-cel provided at least 12 consecutive months of freedom from severe pain crises for patients with sickle cell disease in a mid-stage trial, setting the stage for an FDA application as early as this year.
Bayer’s Sunharbo has secured the first FDA approval of its class, establishing a regulatory pathway for other factor XIa inhibitors, such as Bristol Myers Squibb and Johnson and Johnson’s milvexian.
Having linked its glucagon/GLP-1 candidate to a significant reduction in heavy drinking days, Altimmune is preparing to speak to the FDA in December about the design of a Phase 3 program.
Compass Pathways has completed an FDA filing for COMP360, with an expected launch in the first half of next year. The update follows a reported regulatory submission for Usona Institute’s own psilocybin candidate for depression last Monday.
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.
SK pharmteco’s Tatiana Nanda, chief technical officer of SK pharmteco Viral Vector North America discusses what gene therapy sponsors must account for sooner rather than later when preparing for GMP manufacturing.
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
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.
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