Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says
The FDA has reversed its position on Regenxbio's gene therapy for MPS II, allowing a new path for accelerated approval.
Velocity
How fast coverage is spreading — measured hourly from article rate × source diversity. How this works →
The brief
Food and Drug Administration is set to reconsider a Biologics License Application (BLA) for NAVSUNLI, a gene therapy developed by Regenxbio. This decision follows a previous rejection of the treatment, which is intended for patients with MPS II.
Reporting from The Wall Street Journal, Reuters, Stat News, Fierce Biotech, and Yahoo Finance emphasizes the shift in the agency's regulatory stance. Coverage highlights that this development provides a path forward for what could become the first gene therapy approved for this specific rare disease.
Future updates will depend on the BLA resubmission process. Coverage does not yet specify a timeline for the final regulatory decision following this new alignment between the company and the FDA.
Synthesized by headlinez.news from the headlines below under a strict no-invention contract. ✓ fact-checked: all claims supported by sources Updated 5h ago.
Quick answers
What is the purpose of the NAVSUNLI gene therapy?
It is being developed as a potential treatment for MPS II.
Has the FDA previously reviewed this drug?
Yes, the FDA had previously rejected the therapy before this recent reversal.
What is the current status of the application?
Regenxbio and the FDA have aligned on a path forward for a BLA resubmission seeking accelerated approval.
Coverage (5)
- US FDA reverses course on Regenxbio's rare-disease gene therapy, backs accelerated approval bid Reuters · 2d ago
- In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy Fierce Biotech · 2d ago
- REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II Yahoo Finance · 2d ago
- FDA reverses course on Regenxbio’s childhood gene therapy after rejection statnews.com · 2d ago
- Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says WSJ · 2d ago
Topics
Related trends
Sangamo to put ‘substantially all’ assets for sale in bankruptcy, lines up Lilly and Astellas as buyers
Gene therapy pioneer Sangamo Therapeutics files for bankruptcy, with Lilly and Astellas racing to acquire its assets
Popular Frozen Foods Recalled Nationwide Due to Plastic Contamination
MorningStar Farms has issued a nationwide recall for specific frozen plant-based products following reports of potential plastic contamination.
Blood pressure medication recalled nationwide: FDA - WGN-TV
The FDA has issued a nationwide recall for more than 11,000 bottles of blood pressure medication due to potential ineffectiveness.
HHS Launches Unprecedented Department-Wide Effort to Restore American Leadership in Clinical Trials
U.S. overhauls clinical trials to reclaim global biotech dominance amid China’s rise