FDA grants Regenxbio accelerated approval pathway for Duchenne gene therapy

Regenxbio has received FDA alignment for an accelerated approval pathway for its AFFINITY DUCHENNE trial, advancing RGX-202, a gene therapy for Duchenne muscular dystrophy into its next phase.

The FDA’s decision supports RGX-202 as a potential breakthrough therapy, allowing Regenxbio to expedite clinical development while addressing critical unmet needs for Duchenne patients. The trial will evaluate microdystrophin expression and functional improvements in 30 ambulatory patients aged one and older.

Phase I/II data showed positive outcomes, including functional gains exceeding natural history benchmarks and strong microdystrophin expression in treated patients. No serious safety concerns have been reported at either dose level.

Regenxbio’s RGX-202 is the only late-stage gene therapy for Duchenne incorporating a differentiated microdystrophin construct with the CT domain, offering a promising solution for this severe and progressive disease.