Hunter syndrome
Coverage of Hunter syndrome in the Nexus archive.
- STAT+: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval
Regenxbio announced it will submit its Duchenne muscular dystrophy gene therapy for accelerated approval, despite the FDA previously requesting an additional trial. The FDA appears to be reversing recent rejections of gene therapies, including a Hunter syndrome treatment reconsidered after key officials, Marty Makary and Vinay Prasad, left the agency.
- FDA reconsiders rejected rare-disease gene therapy
The FDA has agreed to reconsider a gene therapy for Hunter syndrome developed by Regenxbio after initially rejecting it due to concerns about clinical trial design. The agency is now open to reviewing existing data rather than requiring new trials, reflecting a broader trend in regulatory flexibility for rare-disease treatments.
- STAT+: FDA reverses course on Regenxbio’s childhood gene therapy after rejection
The FDA will reconsider approving Regenxbio’s gene therapy for a rare childhood brain disorder, mucopolysaccharidosis type II (MPS II), after initially rejecting it four months ago. This follows recent FDA reversals, including allowing UniQure to resubmit a gene therapy application for Huntington’s disease previously rejected by former commissioner Marty Makary.