One-Time Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has granted Fast Track designation to RGX-202 for the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder that results in progressive muscle weakness.

In patients with DMD, the absence of functional dystrophin protein caused by mutations in the DMD gene results in cell damage during muscle contractions leading to cell death, inflammation, and fibrosis of muscle tissues. RGX-202 is an investigational 1-time gene therapy designed to use the AAV8 vector to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal domain.

The safety, tolerability and clinical efficacy of a one-time intravenous dose of RGX-202 in pediatric patients 4 to 11 years of age with DMD is currently being evaluated in the phase 1/2 AFFINITY DUCHENNE trial (ClinicalTrials.gov Identifier: NCT05693142).

“Fast Track designation, along with our capabilities to conduct our clinical trials using commercial-scale cGMP material, will further support the efficient development of RGX-202 from clinic to commercial readiness,” said Kenneth T. Mills, President, and CEO of REGENXBIO. “RGX-202 is a key part of our ‘5×25’ strategy, and we look forward to continuing to work closely with the FDA and the Duchenne community as we advance a highly differentiated product candidate developed with the potential to make a meaningful difference for patients. We look forward to reporting initial data from our clinical trial of RGX-202 in the second half of this year.” 

This article originally appeared on MPR