After hours, Sarepta surged over 33%! Its DMD gene therapy Elevidys approved for expanded indications

According to S Wisdom Finance APP, biotechnology company Sarepta Therapeutics (SRPT.US) surged more than 33% in after-hours trading on Thursday. On the news front, the U.S. Food and Drug Administration (FDA) approved the expansion of indications for the gene therapy Elevidys, developed jointly by Sarepta and Roche, to include Duchenne muscular dystrophy (DMD) patients aged at least 4 years old. FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory DMD patients depends on the results of subsequent confirmatory trials.

Under the accelerated approval pathway, Sarepta has committed to conducting and submitting the results of a randomized controlled trial to validate and confirm the clinical benefits of Elevidys in non-ambulatory DMD patients. The ENVISION (SRP-9001-303) trial is ongoing, which is a global, randomized, double-blind, placebo-controlled Phase 3 study targeting non-ambulatory and older ambulatory DMD patients, aiming to serve as the confirmatory trial required post-approval.

Data shows that in June last year, Elevidys, as the world's first one-time gene therapy for DMD, received FDA approval for marketing, used to treat DMD children aged 4-5 who can walk independently.

Elevidys is a recombinant gene therapy that packages the transgene expressing micro-dystrophin into an AAV viral vector. Through a single intravenous injection, the patient's muscles generate a recombinant protein with partial anti-dystrophin function, which can be effective for patients carrying any type of DMD pathogenic gene mutation. Roche reached a $2.85 billion research and development partnership with Sarepta in 2019 to jointly develop this gene therapy