Sarepta Therapeutics surges over 45% pre-market as FDA approves Elevidys gene therapy for Duchenne muscular dystrophy (DMD) treatment.

Sarepta Therapeutics surges over 45% pre-market as FDA approves Elevidys gene therapy for Duchenne muscular dystrophy (DMD) treatment.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has seen a significant surge in its stock price, rising by over 45% in pre-market trading following the U.S. Food and Drug Administration's (FDA) approval of its gene therapy, ELEVIDYS, for the treatment of Duchenne muscular dystrophy (DMD). The approval marks a significant milestone for Sarepta and the broader biotechnology sector.

ELEVIDYS is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy by delivering a transgene that codes for the targeted production of dystrophin protein. The FDA's decision to approve ELEVIDYS comes after a comprehensive review of the safety data in the ambulatory population, which included a case of an 8-year-old in Brazil whose death was deemed unlikely to be related to treatment with ELEVIDYS by Brazilian health authorities.

The FDA's investigation concluded that the death was unrelated to treatment with ELEVIDYS, and Sarepta was notified that it could resume shipments of ELEVIDYS to sites of care for treatment of ambulatory patients with Duchenne. Sarepta CEO Doug Ingram stated, "We are very pleased that FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of ELEVIDYS for ambulatory patients."

The approval of ELEVIDYS underlines the FDA's commitment to addressing the needs of patients with rare diseases. Sarepta and the FDA will continue to collaborate on the safety labeling process and risk-mitigation approach for non-ambulatory patients, who remain on pause pending further discussions.

Investors and financial professionals are closely watching Sarepta's progress, as the approval of ELEVIDYS could significantly impact the company's revenue and market position. The stock surge is a reflection of the market's optimism about the potential of gene therapy to treat rare and debilitating diseases.

References:
[1] https://investorrelations.sarepta.com/news-releases/news-release-details/fda-informs-sarepta-it-recommends-sarepta-remove-its-pause-and