https://www.live-feeds.com/feed/exclusive-fda-gives-third-rare-disease-drug-another-shot-regenxbio-saysContinuously updated, source-cited coverage. https://www.live-feeds.com/feed/exclusive-fda-gives-third-rare-disease-drug-another-shot-regenxbio-sayshttps://www.live-feeds.com/feed/exclusive-fda-gives-third-rare-disease-drug-another-shot-regenxbio-says#u20613Wed, 01 Jul 2026 22:13:52 +0000The FDA allowed Regenxbio to resubmit its application for the accelerated approval of NAVSUNLI. This therapy targets Mucopolysaccharidosis Type II. The agency's shift in evidence requirements for rare disease drugs may also benefit other biotech firms.What's confirmed:The FDA reversed its rejection of Regenxbio's gene therapy for Hunter syndrome.Regenxbio can resubmit its application for accelerated approval.The FDA also cleared uniQure to file or refile for approval of a gene therapy.Regenxbio will receive $100 million from AbbVie after dosing the first patient in the Phase IIb/III https://www.live-feeds.com/feed/exclusive-fda-gives-third-rare-disease-drug-another-shot-regenxbio-sayshttps://www.live-feeds.com/feed/exclusive-fda-gives-third-rare-disease-drug-another-shot-regenxbio-says#u14702Fri, 26 Jun 2026 20:02:45 +0000The FDA has reversed its rejection of Regenxbio's gene therapy for Hunter syndrome, allowing the company to resubmit its application for accelerated approval. This is the latest in a series of reversals for rare disease treatments. Regenxbio expects to meet with the FDA in July and resubmit its Biologics License Application (BLA) in Q3 2026. The therapy, called NAVSUNLI, is a potential treatment for Mucopolysaccharidosis Type II (MPS II).What's confirmed:The FDA will reconsider Regenxbio's gene therapy for Hunter syndrome.Regenxbio will resubmit its Biologics License Application (BLA