RGNX reports positive pivotal Phase III data for RGX-202, targeting potential accelerated FDA approval in 2027 for Duchenne muscular dystrophy.
Regenxbio announced positive top-line results from the pivotal Phase III trial of RGX-202, its investigational gene therapy for Duchenne muscular dystrophy. The therapy met its primary endpoint, positioning it for a potential accelerated approval pathway in 2027.
The trial results mark a critical milestone for RGX-202, which aims to be a best-in-class treatment for the rare genetic disorder. Management highlighted the data as a key step toward addressing an unmet medical need in the Duchenne community.
Shares of RGNX rose in pre-market trading following the announcement, reflecting investor optimism about the therapy’s commercial prospects and regulatory timeline.