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REGENXBIO Receives FDA Fast Track Designation for RGX-202, a Novel Gene Therapy Candidate for the Treatment of Duchenne Muscular Dystrophy
RGX-202 is a potential one-time AAV Therapeutic for the treatment of Duchenne and includes an optimized transgene for a novel microdystrophin and REGENXBIO's
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-202 is a potential one-time AAV Therapeutic for the treatment of Duchenne and includes an optimized transgene for a novel microdystrophin and REGENXBIO's proprietary NAV® AAV8 vectorCommercial-scale cGMP material from the REGENXBIO Manufacturing Innovation Center to be used in the clinical trialAFFINITY DUCHENNE™ Phase I/II trial of RGX-202 is active and recruiting patients; anticipated to report initial trial data in the second half of 2023 ROCKVILLE, Md., April 11, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. 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).\nFast Track designation aims to facilitate the 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 Orphan Drug Designation and Rare Pediatric Disease Designation.\n\"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 '5x25' 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.\" \n\"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.\"\nRGX-202 Clinical ProgramIn January, REGENXBIO announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne is now active and recruiting patients. AFFINITY DUCHENNE is...