SRP-9001's PDUFA date is set for May 23, 2023.
The FDA has accepted and granted priority review to Sarepta Therapeutics’ biologics license application (BLA) for SRP-9001, an investigative gene therapy for treating Duchenne muscular dystrophy (DMD), with a PDUFA date set for May 29, 2023.1
“We are delighted to announce that the FDA has accepted Sarepta’s BLA for SRP-9001 for filing and priority review,” Doug Ingram, president and chief executive officer, Sarepta Therapeutics, said in a statement.1 “Duchenne is a relentlessly degenerative and invariably fatal disease, robbing children of muscle and function hourly and daily. Our BLA submission for an accelerated approval, along with the FDA’s acceptance of that BLA for filing and review, is a tremendously important milestone in our effort to bring a potentially life-changing gene therapy to Duchenne patients as rapidly as possible and we look forward to working with the FDA through the review process.”
SRP-9001 is developed in partnership with Roche and is designed to express microdystrophin to combat the deleterious effects of the dysfunctional dystrophin produced in DMD. It has been granted fast track, rare pediatric, and orphan drug designations in the US as well as orphan drug status in the EU, Switzerland, and Japan. The BLA was submitted using the accelerated approval pathway in September 2022.2
SRP-9001 has demonstrated safety and efficacy in over 80 treated patients so far, with follow-up of up to 4 years post-treatment. The most recent data were presented from the ENDEAVOR study (Study SRP-9001-103; NCT04626674) in July 2022 and demonstrated an improvement of 4 points on the North Star Ambulatory Assessment (NSAA) from pre-therapy baselines in 20 participants at 52 weeks, as well as a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement compared to a propensity-weighted external control group (P <.0001).3
READ MORE: EMBARK Study Evaluates DMD Gene Therapy
Longer follow-up data from 4 patients were presented from Study SRP-9001-101 (NCT03375164) and showed a 7-point improvement above pre-treatment baselines on the NSAA, a 9.9-point unadjusted means and a 9.4-point least squared means versus a propensity-weighted external control (P = .0125) at 4 years. An integrated analysis of 52 patients across studies SRP-9001-101, SRP-9001-102 (NCT03769116), and ENDEAVOR also showed that at 1 year, patients treated with SRP-9001 at the target dose improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (P <.0001).
“Every hour of every day, this ruthless disease, Duchenne, robs thousands of children in the United States of muscle as it steals their future from them. Sarepta’s BLA submission for an accelerated approval of SRP-9001 is a significant milestone in our quest to intervene with urgency on behalf of the children we serve,” Ingram said in a previous statement.2 “If approved, SRP-9001 will be the first gene therapy available for Duchenne patients. We are enormously grateful to the courageous families who have participated in the SRP-9001 trials and to the participating clinical investigators and experts who have guided us and played a crucial part in reaching this milestone.”