Clinical trial update written by Defeat Duchenne Canada Research Volunteer, Omar Sheikh
REGENXBIO has concluded the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial for the RGX-202 therapy. This is, however, not the end of the full trial.
Crucially, the company continues to enroll ambulatory patients with Duchenne aged 1 year and above in the confirmatory trial.
The company expects to announce their pivotal trial data in early Q2 2026. They will also apply for U.S. FDA approval in mid-2026. REGENXBIO expects success and has started making RGX-202 in preparation for commercial release.
As a reminder, the gene therapy RGX-202 seeks to deliver a smaller version of dystrophin (microdystrophin) to muscle and the heart. In May 2025, REGENXBIO released results on RGX202’s ability to produce measurable dystrophin protein in a safe manner. Patients experienced side effects often associated with gene therapy but are manageable and reversible. These include nausea, vomiting, and fatigue.
Defeat Duchenne Canada is proactively following up with REGENXBIO for details relevant to Canadian patients.
Please see the below press release for more details.
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