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Regenxbio faces regulatory setback as FDA places clinical hold on RGX-121 gene therapy for Hunter syndrome

The U.S. Food and Drug Administration (FDA) placed a clinical hold on Regenxbio's RGX-121 gene therapy, which is being developed to treat Hunter syndrome. The hold was triggered by the discovery of small, asymptomatic nodules or cystic masses in the spines of five participants in the CAMPSIITE study. While investigators classified these findings as non-serious and likely benign, the company stated there is no clinical or pathological evidence to confirm their exact cause. Regenxbio and its partner NS Pharma are currently evaluating the data to determine the next steps for the therapy's development. This regulatory setback follows a previous rejection of the marketing application in February, which was then expected to resubmit in the third quarter of this year. However, the company now no longer expects to resubmit the application in the near term. Curran Simpson, CEO of Regenxbio, noted that the findings appear unique to the Hunter Syndrome program and require further analysis to assess the benefit-risk profile of the drug.

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