Exclusive | FDA Will Reverse Rejection of Third Rare-Disease Drug, Regenxbio Says
The FDA is reversing its rejection of Regenxbio's gene therapy for Hunter syndrome, marking the third such reversal for rare-disease drugs.
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📍 How it ended
The FDA reversed its previous rejection of Regenxbio's rare-disease gene therapy for Hunter syndrome. The agency backed a bid for accelerated approval, allowing Regenxbio to resubmit the BLA for NAVSUNLI.
Epilogue added 23d ago, after coverage quieted.
The brief
The FDA will allow Regenxbio to resubmit its gene therapy, NAVSUNLI, intended for the treatment of MPS II (Hunter syndrome). This move follows a previous rejection of the childhood gene therapy.
Coverage from the Wall Street Journal, Stat News, Endpoints News, and Stock Titan emphasizes that this represents a continuing 'about-face' by the FDA regarding rare-disease drugs. The Wall Street Journal reports this is the third rare-disease drug rejection the agency has reversed.
Future developments center on the resubmission of the therapy for MPS II. Additionally, Seeking Alpha highlights ongoing attention toward Regenxbio's Duchenne data.
Synthesized by Archynetys from the headlines below under a strict no-invention contract. ✓ fact-checked: all claims supported by sources Updated 26d ago.
Quick answers
What drug is being resubmitted to the FDA?
Regenxbio is resubmitting NAVSUNLI, a gene therapy for MPS II (Hunter syndrome).
How many rare-disease drug rejections has the FDA reversed?
According to the Wall Street Journal, this is the third rare-disease drug rejection the FDA will reverse.
What other data is currently being monitored for Regenxbio?
Coverage from Seeking Alpha indicates that the company's Duchenne data remains a point of focus.
Coverage (13)
- RGNX Retail Investors See Hope As FDA Reopens Door For Regenxbio’s Hunter Syndrome Therapy Yahoo Finance · 26d 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 · 26d ago
- FDA reconsiders rejected rare-disease gene therapy Baton Rouge Business Report · 26d ago
- US FDA reverses course on Regenxbio's rare-disease gene therapy, backs accelerated approval bid Reuters · 26d ago
- REGENXBIO to resubmit application for Hunter gene therapy after FDA’s most recent pivot BioSpace · 26d ago
- In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy Fierce Biotech · 26d ago
- Reuters US Domestic News Summary Devdiscourse · 26d ago
- US FDA Reverses Course on Regenxbio's Rare-Disease Gene Therapy, Backs Accelerated Approval Bid U.S. News & World Report · 26d ago
- Regenxbio to resubmit Hunter syndrome gene therapy as FDA rare disease about-face continues Endpoints News · 26d ago
- REGENXBIO gets FDA path to resubmit NAVSUNLI for MPS II Stock Titan · 26d ago
- REGENXBIO’s Duchenne Data Keeps Me Bullish, But There’s Less Room For Setbacks Now (RGNX) Seeking Alpha · 26d ago
- FDA reverses course on Regenxbio’s childhood gene therapy after rejection statnews.com · 26d ago
- Exclusive | FDA Will Reverse Rejection of Third Rare-Disease Drug, Regenxbio Says WSJ · 26d ago
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