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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients

June 25, 2024

Defeat Duchenne Canada is pleased to share news that this morning REGENXBIO issued a press release announcing they are expanding their AFFINITY DUCHENNE Phase 1/2 trial of RGX-202 to include boys 1–3 years old.  This expansion to include younger boys means advancing RGX-202 as a potential gene therapy treatment option for boys 1-11 years old with Duchenne. These trials are currently being held only in the United States.

Highlights include:

  • Data from this younger cohort is part of our pivotal plans and a Biologics License Application (BLA) filing for seeking a broad label when formally requesting approval to market in the United States.
  • Sharing their End-of-Phase II meeting with the FDA will occur in late July to finalize design of the pivotal program.  Based on recent commercial landscape, confirmed accelerated approval pathway remains available, given ongoing unmet need and RGX-202’s differentiated design.
  • REGENXBIO remains on track to start the pivotal trial later this year (late Q3/early Q4). Defeat Duchenne Canada will continue to advocate to have Canadian Sites in the pivotal trial.  No formal decision at this time.

Read the full press release below:

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