Why did the FDA place a clinical hold on Regenxbio's gene therapy programs?

The FDA placed a clinical hold on Regenxbio's RGX-111 and RGX-121 gene therapy programs due to safety concerns, specifically the discovery of a brain tumor in a trial participant.

What specific gene therapies were affected by the FDA's clinical hold?

The FDA's clinical hold affected Regenxbio's experimental gene therapies RGX-111, for Hurler syndrome, and RGX-121, for Hunter Syndrome.

Were there other reported tumors in patients treated with RGX-111 or RGX-121?

No, Regenxbio reported that no other tumors have been observed in the nine other patients treated with RGX-111 or among the 32 patients treated with RGX-121.

Home / Health / Gene Therapy Halt: Tumor Fears Halt Childhood Disease Trial

Gene Therapy Halt: Tumor Fears Halt Childhood Disease Trial

28 Jan

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Summary

FDA halts two Regenxbio gene therapy programs due to safety concerns.
A brain tumor was discovered in a child who received experimental RGX-111.
No other tumors reported in patients treated with RGX-111 or RGX-121.

Gene Therapy Halt: Tumor Fears Halt Childhood Disease Trial

Regenxbio's stock experienced a significant decline following the U.S. Food and Drug Administration's decision to place a clinical hold on two of its gene therapy programs. These programs were under development for rare childhood diseases.

The experimental therapy RGX-111, intended for Hurler syndrome, was halted after a brain tumor was identified in a five-year-old trial participant. This tumor was found during a routine scan approximately four years after the child received the treatment. The company noted that this is the only tumor reported among nine patients treated with RGX-111.

Additionally, RGX-121, which targets Hunter Syndrome, was also put on clinical hold. The FDA cited potential shared risks between the two programs. Regenxbio expressed surprise at this decision, especially as investigations into the RGX-111 incident continue. Importantly, 32 patients treated with RGX-121 have not experienced similar tumor reports.