FDA
Compass Pathways revealed that an FDA filing for COMP360 will be complete “in the next two weeks,” with an expected drug launch in the first half of next year. The update follows a reported regulatory submission for Usona Institute’s own psilocybin candidate for depression on Monday.
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 With the FDA’s latest draft guidance, it’s coming to fruition.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.
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.
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.
CROs cite faster startup timelines, regulatory predictability and tax incentives as reasons for growing interest in Australia. State investments in manufacturing, translational research and advanced therapies aim to expand that advantage into a broader biotech ecosystem. The question remains whether Australia can support drug development at broader scale.
AbbVie is eyeing regulatory filings in 2027 for the skin disease drug lutikizumab after clearing multiple endpoints in a late-stage trial, but the results have disappointed some analysts, raising questions about its commercial prospects.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
PRESS RELEASES