Regenxbio Gene Therapy Candidate Gets Fast Track Status for Duchenne Muscular Dystrophy
The FDA has granted Fast Track designation to Regenxbio’s RGX-202 candidate as a potential one-time gene therapy treatment for Duchenne muscular dystrophy.
The disorder, which is characterized by progressive muscular weakness, is caused by a genetic mutation that affects the production of dystrophin, resulting in cell damage during muscle contraction. RGX-202 introduces a novel gene to make muscles resistant to the contraction caused by the lack of dystrophin.
The gene therapy candidate is being evaluated in a phase 1/2 clinical trial, in which six patients ages four to 11 years are expected to enroll for a one-time intravenous dose of the medication.
The FDA grants the Fast Track designation to facilitate development and expedite review of new therapeutics to treat serious or life-threatening conditions and to address unmet medical needs.
The agency previously granted RGX-202 Orphan Drug and Rare Pediatric Disease designations.
April 14, 2023
https://www.fdanews.com/
The disorder, which is characterized by progressive muscular weakness, is caused by a genetic mutation that affects the production of dystrophin, resulting in cell damage during muscle contraction. RGX-202 introduces a novel gene to make muscles resistant to the contraction caused by the lack of dystrophin.
The gene therapy candidate is being evaluated in a phase 1/2 clinical trial, in which six patients ages four to 11 years are expected to enroll for a one-time intravenous dose of the medication.
The FDA grants the Fast Track designation to facilitate development and expedite review of new therapeutics to treat serious or life-threatening conditions and to address unmet medical needs.
The agency previously granted RGX-202 Orphan Drug and Rare Pediatric Disease designations.
April 14, 2023
https://www.fdanews.com/
