On Tuesday, Sarepta Therapeutics, Inc. (NASDAQ: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.
Read Next:
UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets.
Get the latest stock analysis from Benzinga?
This article Sarepta Therapeutics Reports Patient Death Post Elevidys Gene Therapy Treatment, Stock Plunges originally appeared on Benzinga.com
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.