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.
📍 Where it landed
The FDA reversed its earlier rejection and agreed to reconsider Regenxbio’s accelerated approval request for its gene therapy. The company announced alignment with the agency on a path forward for the resubmission of its biologics license application.
Coverage of the story has since quieted without further updates on the outcome.
Epilogue added 49d ago, after coverage quieted.
The reporting (5)
- US FDA reverses course on Regenxbio's rare-disease gene therapy, backs accelerated approval bid Reuters · 51d ago
- In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy Fierce Biotech · 51d 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 · 51d ago
- FDA reverses course on Regenxbio’s childhood gene therapy after rejection statnews.com · 51d ago
- Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says WSJ · 51d ago
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 49d 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.
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