Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says
The FDA is allowing Regenxbio to resubmit its gene therapy for Hunter syndrome, marking a reversal of a previous rejection.
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The brief
Regenxbio has been granted a path to resubmit its gene therapy, known as NAVSUNLI, for the treatment of Hunter syndrome (MPS II). This follows a previous rejection by the FDA regarding the childhood treatment.
Coverage from the Wall Street Journal, Endpoints News, Stat News, Stock Titan, and Seeking Alpha highlights the regulatory shift. Reports emphasize that this marks a broader trend of the FDA reconsidering previous decisions for rare-disease drugs.
Regenxbio is expected to move forward with the resubmission process for the drug. Market observers note that the company faces heightened pressure to deliver results regarding its wider pipeline, including ongoing work in Duchenne muscular dystrophy.
Synthesized by PULSE from the headlines below under a strict no-invention contract. ✓ fact-checked: all claims supported by sources Updated 120d ago.
Quick answers
What is the status of the Hunter syndrome gene therapy?
The FDA has granted Regenxbio permission to resubmit its application for NAVSUNLI following a prior rejection.
Which condition is the therapy intended to treat?
The therapy is intended for Hunter syndrome, also referred to as MPS II.
What other conditions is Regenxbio researching?
Coverage indicates the company is also working on treatments for Duchenne muscular dystrophy.
Coverage (12)
- REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II Yahoo Finance · 120d ago
- FDA reconsiders rejected rare-disease gene therapy Baton Rouge Business Report · 120d ago
- US FDA reverses course on Regenxbio's rare-disease gene therapy, backs accelerated approval bid Reuters · 120d ago
- REGENXBIO to resubmit application for Hunter gene therapy after FDA’s most recent pivot BioSpace · 120d ago
- In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy Fierce Biotech · 120d ago
- Reuters US Domestic News Summary Devdiscourse · 120d ago
- US FDA Reverses Course on Regenxbio's Rare-Disease Gene Therapy, Backs Accelerated Approval Bid U.S. News & World Report · 120d ago
- Regenxbio to resubmit Hunter syndrome gene therapy as FDA rare disease about-face continues Endpoints News · 120d ago
- REGENXBIO gets FDA path to resubmit NAVSUNLI for MPS II Stock Titan · 120d ago
- REGENXBIO’s Duchenne Data Keeps Me Bullish, But There’s Less Room For Setbacks Now (RGNX) Seeking Alpha · 120d ago
- FDA reverses course on Regenxbio’s childhood gene therapy after rejection statnews.com · 120d ago
- Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says WSJ · 120d ago
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