Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy (DMD), following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial.

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy for Duchenne muscular dystrophy.