The FDA granted Regenxbio RGX-202, a prospective gene therapy for Duchenne Muscular Dystrophy, a Fast Track designation.
Regenxbio announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne).
An FDA Fast Track designation helps facilitate development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
Therapies granted this designation are given the opportunity for more frequent interactions with the FDA and may qualify for priority review. The FDA has granted RGX-202 an Orphan Drug Designation and Rare Pediatric Disease Designation.
“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 chief executive officer 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.”
In January, Regenxbio announced that the Phase I/II Affinity Duchenne trial of RGX-202 for the treatment of Duchenne was active and recruiting patients. Affinity Duchenne is a multicenter, open-label dose evaluation and dose expansion clinical study to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne.
Additionally, Regenxbio is recruiting patients in the Affinity Beyond trial, an observational screening study to evaluate the prevalence of AAV8 antibodies in patients with Duchenne up to 12 years of age. Information collected in this study may be used to identify potential participants for the Affinity Duchenne trial and potential future trials of RGX-202.
“We are pleased that the FDA has granted Fast Track designation for RGX-202,” said Debra Miller, founder and CEO of CureDuchenne. “Accelerating the development of medicines for Duchenne, especially potential one-time gene therapies like RGX-202, is critical for this community.”
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