REGENXBIO’s Hunter syndrome gene therapy hit by FDA hold, stymieing resubmission plans
RegulatoryRGX-121Negative
AI Analysis
Summary
FDA placed a clinical hold on REGENXBIO's RGX-121 gene therapy for Hunter syndrome following detection of nodules or cystic masses in the spines of five patients in the registrational trial, leading the company to abandon near-term resubmission plans after a prior rejection in February.
Clinical Trial Data
Phase
registrational
Primary Endpoint
Not Met
Patients
5
Outcome Details
FDA placed clinical hold on RGX-121 after small nodules or cystic masses detected in spines of five patients in registrational trial
Importance:9/10
Sentiment:
-0.95
gene therapyclinical holdFDA actionHunter syndromesafety concernstrial suspension
Related Companies
Read the original article
Published by BioSpace on August 24, 2026 1:44 PM