Regenxbio reports key gene therapy results, moves toward FDA accelerated clearance for Duchenne

Regenxbio said its experimental gene therapy RGX-202 met the primary endpoint in a pivotal Duchenne muscular dystrophy trial, positioning the company to file for accelerated FDA clearance.

In the study, 28 of 30 participants who received muscle biopsies showed at least 10% of normal microdystrophin levels three months after dosing. Nine patients with a year of follow-up also showed statistically significant gains on several motor function tests.

Two serious adverse events were reported – one case of heart inflammation and one of asymptomatic liver injury – both were managed and resolved within weeks, and average liver enzyme levels stayed within normal limits. CEO Curran Simpson expressed optimism that the FDA will apply flexibility for rare-disease therapies as the company prepares its submission.