FDA lifts pause on Sarepta’s Duchenne gene therapy for ambulatory patients

CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday that the U.S. Food and Drug Administration has authorized the company to resume shipments of its Duchenne muscular dystrophy gene therapy ELEVIDYS for ambulatory patients. The news comes as the company’s stock trades near its 52-week low, having declined over 90% year-to-date according to InvestingPro data.

The company had voluntarily paused shipments last week at the FDA’s suggestion to allow for a safety review. The FDA has now completed what Sarepta described as a "rapid and comprehensive" assessment of available safety information.

Shipments to treatment centers for ambulatory patients will resume immediately, according to the company’s statement. However, the pause remains in effect for non-ambulatory patients pending further discussions with regulators.

The FDA’s review included investigation of an 8-year-old patient death in Brazil, which authorities have concluded was unrelated to ELEVIDYS treatment.

"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," said Doug Ingram, chief executive officer of Sarepta.

The company indicated it will continue working with the FDA to update safety labeling for the therapy and develop a risk-mitigation approach for non-ambulatory patients.

ELEVIDYS is a single-dose gene therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy through delivery of a transgene that codes for micro-dystrophin production in skeletal muscle.

The therapy received accelerated approval for non-ambulatory patients, which remains contingent upon verification of clinical benefit in confirmatory trials.

Based on a press release statement from Sarepta Therapeutics, the therapy is the only approved gene therapy for Duchenne, a progressive and ultimately fatal rare disease. Investors should note that the company is scheduled to report its next earnings on July 30, with InvestingPro data showing 7 analysts have revised their earnings expectations downward for the upcoming period. For deeper insights into Sarepta’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.

In other recent news, Sarepta Therapeutics has faced several notable developments. The company reported significant challenges, including three patient deaths within its gene therapy portfolio and the removal of its Elevidys product from the U.S. market. Additionally, the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a negative recommendation for EU approval of Elevidys. These setbacks have influenced analyst opinions, with H.C. Wainwright reiterating a Sell rating and setting a $0 price target for Sarepta. Barclays also downgraded Sarepta to Underweight, reducing its price target to $10, citing Elevidys concerns. Similarly, JPMorgan downgraded the stock to Underweight, removing its previous $16 price target. In contrast, Jefferies maintained a Buy rating but lowered its price target to $35, referencing Roche’s reduced ex-US sales guidance for Elevidys. On a positive note, Sarepta facilitated a $100 million milestone payment to Arrowhead Pharmaceuticals after reaching enrollment targets in a clinical study. These developments reflect the current landscape for Sarepta Therapeutics amidst ongoing challenges and strategic adjustments.

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