FDA places clinical hold on Regenxbio RGX-121 gene therapy for Hunter syndrome after spine MRI findings - RGNX News | RalliesFDA places clinical hold on Regenxbio RGX-121 gene therapy for Hunter syndrome after spine MRI findings
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RGNX• FDA places clinical hold on RGX-121 after spine MRI findings
- FDA imposed a clinical hold on RGX-121 for MPS II following asymptomatic spine MRI findings in five CAMPSIITE participants.
- Regenxbio does not expect to resubmit the RGX-121 BLA in the near term.
- Findings involved small nodules or cystic masses on spine MRI, seen about three to six years post-dose; investigators deemed them nonserious.
- The company is evaluating additional imaging and longer-term follow-up data, incorporating FDA feedback into next steps for RGX-121.
