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Gene Therapy Halt: Tumor Fears Halt Childhood Disease Trial
28 Jan
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.
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.
