Trial Profile
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
Status:
Active, no longer recruiting
Phase of Trial:
Phase III
Latest Information Update: 21 Jun 2024
Price :
$35
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At a glance
- Drugs Delandistrogene moxeparvovec (Primary)
- Indications Duchenne muscular dystrophy
- Focus Registrational; Therapeutic Use
- Acronyms EMBARK
- Sponsors Sarepta Therapeutics
- 20 Jun 2024 According to a Sarepta Therapeutics media release, company will host an investor conference call on June 21, 2024, at 8:30 a.m. ET to discuss this update.
- 20 Jun 2024 According to a Sarepta Therapeutics media release, company announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. The FDA granted traditional approval for ambulatory patients and the FDA granted accelerated approval for non-ambulatory patients.
- 22 Dec 2023 According to a Sarepta Therapeutics media release, the company has submitted an efficacy supplement to the Biologics License Application (BLA) to US FDA with a request for Priority Review for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows [ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene, supported by results from EMBARK and ENDEAVOR trial.