Regenxbio’s Duchenne Gene Therapy Shows Success in Trial, FDA Submission Next

Regenxbio announced on Thursday that its experimental gene therapy for Duchenne muscular dystrophy achieved sufficiently high levels of a miniaturized muscle protein, which is deficient in the fatal neuromuscular disease. The milestone paves the way for the company to submit its data to the U.S. Food and Drug Administration (FDA).

The biotech firm aims to develop a Duchenne gene therapy that outperforms Sarepta Therapeutics’ Elevidys, a treatment already on the market but facing significant safety concerns. These concerns escalated after two recipients died from liver failure, raising questions about Elevidys’ risk profile.

“I think our data checks every single box that you would want for accelerated approval,”

Curran Simpson, CEO of Regenxbio, told STAT.

Regenxbio’s therapy is designed to address the root cause of Duchenne muscular dystrophy by delivering a functional copy of the dystrophin gene, which is mutated or missing in patients. The protein produced by the therapy, known as delandistrogene moxeparvovec, is a truncated but functional version of dystrophin, intended to restore muscle function and slow disease progression.

The company’s announcement follows promising data from its Phase 2b clinical trial, which demonstrated measurable improvements in muscle function and protein expression levels in treated patients. Regenxbio plans to present detailed findings from the trial at an upcoming medical conference.

If approved, Regenxbio’s therapy could offer a safer and more effective alternative to existing treatments, including Elevidys. The company’s submission to the FDA is expected to include comprehensive data from the trial, as well as long-term safety and efficacy results.