FDA grants fast track designation for novel Duchenne muscular dystrophy gene therapy
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Key takeaways:
- FDA previously granted orphan drug, rare pediatric disease designations for RGX-202.
- Phase 1/2 study will examine safety, efficacy, tolerability of one-time IV dose in six children with Duchenne MD.
Regenxbio Inc. announced that the FDA has granted fast track designation for RGX-202, a one-time gene therapy for treatment of Duchenne muscular dystrophy.
According to a release from the company, the FDA previously granted orphan drug designation and rare pediatric disease designation for RGX-202, which is designed to support delivery and expression of genes in heart and skeletal muscle using the NAV AAV8 vector and a muscle-specific promoter.
In January, Regenxbio announced a multicenter, open-label dose evaluation phase 1/2 Affinity Duchenne study, which will examine the safety, efficacy and tolerability of a one-time IV dose of RGX-202. The company plans to enroll six ambulatory patients aged 4 to 11 years with Duchenne, with the potential for an additional six patients in a dose expansion phase after independent review of safety data, the release stated.
“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,” Kenneth T. Mills, president and CEO of Regenxbio, said in the release. “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.”
The Maryland-based biotech company also announced it is recruiting for the Affinity Beyond trial, an observational screening study evaluating the prevalence of AAV8 antibodies in children up to 12 years. Data collected in this study may help identify potential participants for the Affinity Duchenne trial and possible future trials of RGX-202. Regenxbio anticipates reporting initial data from the phase 1/2 clinical trial of RGX-202 in the latter half of 2023.