FDA's official announcements or press releases

Regenxbio's RGX-202 Gene Therapy Shows Positive Results, FDA Approval Pathway Available

Nov 18, 2024, 12:34 PM

Regenxbio has reported promising results from its experimental gene therapy, RGX-202, for treating boys with Duchenne muscular dystrophy. The company presented clinical evidence showing improved muscle function in patients following the treatment. Regenxbio's CEO, Curran Simpson, indicated in an interview with STAT on Sunday that the FDA has communicated that the accelerated approval pathway remains available for this therapy. The positive data from a small trial is expected to support Regenxbio's pursuit of regulatory approval.