Sarepta Therapeutics Reports Patient Death Post Elevidys Gene Therapy Treatment, Stock Plunges

On Tuesday, Sarepta Therapeutics, Inc. SRPT stock is sliding. The company shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.

The company reported a patient death following treatment with Elevidys, having suffered acute liver failure.

Acute liver injury is a known possible side effect of Elevidys and other AAV-mediated gene therapies and is highlighted in the prescribing information.

Also Read: Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development

The company says although it is not a new safety signal and the benefit-risk of Elevidys remains positive, acute liver failure leading to death represents a severity of acute liver injury not previously reported for Elevidys, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.

In addition, testing revealed that this patient had a recent cytomegalovirus (CMV) infection, which the treating physician identified as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis.

Elevidys is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of Elevidys micro-dystrophin in skeletal muscle.

In January, Sarepta Therapeutics released topline results from Part 2 of the EMBARK Phase 3 study of Elevidys.

Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years), crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR) and 10-meter walk/run (10MWR) function tests compared with a pre-specified, propensity-weighted external control group.

In June 2024, the FDA approved the labeled indication for Elevidys to include individuals with Duchenne muscular dystrophy who have a confirmed mutation in the DMD gene and are at least four years old.

Price Action: SRPT stock is down 22.90% at $78.10 during the premarket session at the last check Tuesday.

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