New Medicines

Duchenne muscular dystrophy in ambulatory patients ages 4-7 years


Advanced therapy medicinal product (ATMP)
Sarepta Therapeutics

Development and Regulatory status

Phase II Clinical Trials
Dec 19 · Sarepta announces a licensing agreement granting Roche the exclusive right to launch and commercialise SRP-9001 outside the United States. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the US [3].


Gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Given as a single infusion.
DMD affects about 1 in 3,500 newborn males [1].
Duchenne muscular dystrophy in ambulatory patients ages 4-7 years
Intravenous infusion

Trial or other data

Sep 20 · PII study (NCT03375164) is also known as Study 101 [2].
Sep 20 · PII study (NCT03769116) is also known as Study 102 [2].
Jun 20 · Sarepta announces safety and tolerability data at one year from four DMD clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. In the open-label trial, known as Study 101, four ambulatory participants between the ages of 4 and 7 were treated with an infusion of SRP-9001 at a dose of 2x1014 vg/kg. The therapy was safe and tolerable in all participants over the one-year time period. All adverse events were considered mild or moderate, and there were no serious adverse events or evidence of complement activation. At 12 weeks, muscle dystrophin levels demonstrated a mean of 81.2% muscle fibers expressing micro-dystrophin with a mean intensity at the sarcolemma by immunohistochemistry of 96% compared to normal biopsies. Adjusted for fat and fibrotic tissue, western blot showed a mean expression of 95.8%. All participants had confirmed vector transduction and showed functional improvement on the North Star Ambulatory Assessment scale (NSAA) and reduced creatine kinase (CK) levels that were maintained through one year [3].
Mar 20 · PII study (NCT03769116) is no longer recruiting [2].
Dec 18 · PII study to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2) starts (NCT03769116). 41 boys aged 4 to 7 years will be recruited in the US. Primary outcomes are 1. Incidence of Serious Adverse Events (SAEs) up to Week 144; 2. Incidence of Treatment Emergent Adverse Events (TEAEs); 3. Change From Baseline in Quantity of Microdystrophin Protein Expression Measured by Western Blot from baseline up to Week 12. Collection of these data is due to complete Oct 22 [2].