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REGENXBIO completes RGX-202 dosing in confirmatory study
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REGENXBIO completes RGX-202 dosing in confirmatory study

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  • REGENXBIO (NASDAQ:RGNX) completed dosing in the confirmatory study of RGX-202 for Duchenne muscular dystrophy (DMD).
  • The program supports a planned Biologics License Application (BLA) filing in Q3 2026 under the FDA accelerated approval pathway.
  • The dataset includes safety data from 63 patients and pivotal efficacy data from 30 patients.

REGENXBIO (NASDAQ:RGNX) said it has completed dosing in the confirmatory study of RGX-202, an investigational gene therapy for Duchenne muscular dystrophy, marking completion of its registrational development program.

The company said the data package supporting the program includes safety results from 63 patients and pivotal efficacy data from 30 patients across clinical studies.

REGENXBIO said the results support initiating a Biologics License Application submission in the third quarter of 2026 under the FDA accelerated approval pathway.

The company is targeting potential FDA approval in the second half of 2027, pending regulatory review of the full clinical dataset and application process.

RGX-202 is being developed as a gene therapy for Duchenne muscular dystrophy, a progressive genetic muscle disorder with limited treatment options.

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