After securing an extended regulatory approval, Sarepta Therapeutics, Inc. (NASDAQ: SRPT) witnessed a significant rise in its stock value on the US charts today. As of the latest current-market check, SRPT stock surged by 35.32%, reaching $167.13.
FDA Approves ELEVIDYS’s Expanded Indication
The U.S. Food and Drug Administration (FDA) has authorized an additional indication for ELEVIDYS, Sarepta (SRPT) unveiled in a statement. This expansion covers people with Duchenne muscular dystrophy (DMD) who are at least 4 years old and have a verified mutation in the DMD gene.
For patients who could walk around, the FDA gave regular approval; for patients who couldn’t, it gave expedited approval. The continuation of approval for patients who are not ambulatory will be subject to confirmation of the clinical benefit through a confirmatory investigation. Notably, patients with deletions in DMD gene exons 8 and/or 9 should not use ELEVIDYS.
Significance For The Duchenne Community And Gene Therapy
The expansion of the ELEVIDYS label represents a culmination of years of dedicated research, development, investment, and innovation. This milestone is pivotal for the Duchenne community, providing treatment options for patients aged 4 and above, irrespective of ambulatory status. The approval is a major milestone for the science of gene therapy and illustrates how scientific progress may be used to cure hereditary illnesses.
Ongoing Commitments And Future Trials
Sarepta has agreed to undertake a randomized, controlled trial to confirm the therapeutic benefit of ELEVIDYS in non-ambulatory Duchenne patients, in line with the fast approval procedure. A worldwide, randomized, double-blind, placebo-controlled Phase 3 study called ENVISION is presently under conducted. This study includes non-ambulatory and older ambulatory individuals with Duchenne and aims to fulfill the postmarketing requirements.
Collaboration With Roche
Sarepta and Roche are collaborating to better the future for the Duchenne community under the terms of a partnership agreement that was struck in 2019. With this collaboration, patients’ ability to use their muscles will be preserved. Sarepta is responsible for regulatory approval, commercialization of ELEVIDYS in the U.S., and manufacturing, while Roche handles regulatory approvals and patient access to ELEVIDYS in other regions worldwide.
