REGENXBIO (NASDAQ: RGNX) has completed dosing in the confirmatory study of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy (DMD).
According to REGENXBIO, this milestone positions the company to initiate a Biologics License Application under the accelerated approval pathway in Q3 2026 for a potential approval by the FDA in the second half of 2027.
In a statement, Curran Simpson, president and CEO of REGENXBIO, commented, “Completing the enrollment of our confirmatory study ahead of schedule is a significant milestone that underscores the urgent unmet need in Duchenne and brings us another step closer to advancing RGX-202 towards approval via the accelerated approval pathway. We are encouraged by the recent FDA trends in rare disease development, including our collaborative discussion with FDA regarding our Hunter Syndrome program, demonstrating that the accelerated approval pathway remains available and supported for rare diseases. The RGX-202 pivotal dataset directly aligns with the established accelerated approval criteria: the magnitude of clinical effect seen in functional improvement from baseline, correlation between the biomarker and functional outcomes, and a differentiated safety profile. For all of these reasons, we believe we are well positioned to deliver RGX-202 as the next approved gene therapy for Duchenne patients.”
