THis unexpected rejection by the Food and Drug Administration of a gene therapy for the hemophilia of the thriving disorder has resurfaced across the biotech industry, potentially complicating the path to the market for these one-time treatments.
In deciding not to approve Roctavian, a BioMarin Pharma Gene Therapy for Hemophilia A, the FDA required two additional years of data to reinforce the case that its benefits are sustainable. This is in contrast to the agency’s observed linearity in approving previous one-time therapies for rare diseases, and it may affect the plans of other gene therapy companies in the hopes of gaining FDA approval based on early signs. of promise.
Unlock this article by subscribing to STAT Plus and enjoy your first 30 days for free!
GET STARTED
What is it?
STAT Plus is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life sciences coverage and analysis. Our award-winning team covers news about Wall Street, policy developments in Washington, early breakthroughs in science and clinical trial outcomes, and limitation of health care in Silicon Valley and beyond.
What is included?
- Daily reporting and analysis
- The most comprehensive coverage of the sector from a powerhouse team of reporters
- Newsletters for subscribers
- Daily newsletters to keep you posted on the most important news of the day
- STAT + conversations
- Weekly Opportunities to Chat with Our Reporters and Leading Experts in Live Video Conversations
- Exclusive events in the sector
- Premium access to network subscribers only in the country
- The best reporters in the sector
- The most trusted and well-connected newsroom in the healthcare sector
- And so much more
- Exclusive interviews with leaders, profiles and premium tools, such as our CRISPR Trackr.
