FDA rejects Regenxbio’s Hunter syndrome gene therapy over biomarker doubts
TL;DR
The FDA has rejected Regenxbio’s RGX-121 gene therapy for mucopolysaccharidosis type II (Hunter syndrome), citing concerns about the surrogate cerebrospinal fluid biomarker used to predict longer-term cognitive benefit and casting doubt on the accelerated-approval pathway.
- FDA rejects Regenxbio’s rare-disease gene therapy statnews.com
- FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned' Fierce Biotech
- REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II Yahoo Finance
- FDA rejects Regenxbio’s Hunter syndrome gene therapy Endpoints News
- US FDA declines to approve Regenxbio's gene therapy for rare disease Reuters
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