Regulatory
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.
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 With the FDA’s latest draft guidance, it’s coming to fruition.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.
Saudi Arabia is building the regulatory framework, partnerships infrastructure and IP landscape needed to support a biotech ecosystem, despite regional political instability. Whether it can produce sustainable biotech companies and a resilient manufacturing base for global production remains an open question, but the future looks bright.
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.
In this episode of Denatured, you’ll hear from Jerome Barley, commercial director at Avion Medical and Ryan Klein, vice president, business development, US and APAC at Resolian. We discuss why Australia has become a magnet for early clinical development, and whether its speed, regulatory clarity and tax incentives are pushing it toward a more integrated biotech role.
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