REGENXBIO Inc. announced on November 18, 2024, that its AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, has advanced to the pivotal stage with the first patient dosed. This marks a critical step towards potential market approval.
New positive efficacy and safety data from the Phase I/II portion of the study were also reported, including the first functional data. The initial five participants demonstrated stable or improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests, exceeding external natural history controls.
Pivotal dose participants showed an average improvement of 5.5 points on the NSAA at nine months. RGX-202 was well tolerated with no serious adverse events or adverse events of special interest as of November 1, 2024. A Biologics License Application (BLA) filing is targeted for 2026 using the accelerated approval pathway.
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