FDA nixes Regenxbio Hunter syndrome gene therapy over trial design concerns
TL;DR
The FDA rejected Regenxbio’s RGX-121 for Hunter syndrome, citing unresolved issues in the trial design (defining the patient population), use of a natural history control, and a biomarker surrogate endpoint, with a PDUFA date already pushed from 2025. Regenxbio plans to resubmit with longer-term data. The decision follows a broader context of safety concerns linked to Regenxbio’s RGX-111 and its brain-tumor event, but the FDA noted those issues were not cited as reasons for RGX-121’s rejection. Industry experts emphasize ongoing questions around biomarker-based approvals and vector-related safety, underscoring the urgent need for effective therapies in these ultra-rare MPS diseases.
- FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned' Fierce Biotech
- US FDA declines to approve Regenxbio's gene therapy for rare disease Reuters
- FDA rejects Regenxbio’s rare-disease gene therapy statnews.com
- FDA Denies Regenxbio Therapy in Latest Rare Disease Setback bloomberg.com
- FDA Slams REGENXBIO's MPS II Gene Therapy RGX-121 With CRL CGTLive®
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