On Tuesday, Sarepta Therapeutics, Inc. SRPT inventory is sliding. The corporate shared an replace on Elevidys (delandistrogene moxeparvovec-rokl), the one accredited gene remedy in sufferers with Duchenne muscular dystrophy.
The corporate reported a affected person demise following remedy with Elevidys, having suffered acute liver failure.
Acute liver harm is a recognized doable aspect impact of Elevidys and different AAV-mediated gene therapies and is highlighted within the prescribing data.
Additionally Learn: Strong Biosciences Outpaces Rivals In Duchenne Gene Remedy Growth
The corporate says though it isn’t a brand new security sign and the benefit-risk of Elevidys stays optimistic, acute liver failure resulting in demise represents a severity of acute liver harm not beforehand reported for Elevidys, which thus far has been used to deal with greater than 800 sufferers in medical trials or as a prescribed remedy.
As well as, testing revealed that this affected person had a current cytomegalovirus (CMV) an infection, which the treating doctor recognized as a doable contributing issue. CMV can infect and injury the liver, a situation often known as CMV hepatitis.
Elevidys is a single-dose, adeno-associated virus (AAV)-based gene switch remedy for intravenous infusion designed to deal with the underlying genetic reason for Duchenne muscular dystrophy – mutations or modifications within the DMD gene that end result within the lack of dystrophin protein – via the supply of a transgene that codes for the focused manufacturing of Elevidys micro-dystrophin in skeletal muscle.
In January, Sarepta Therapeutics launched topline outcomes from Half 2 of the EMBARK Section 3 research of Elevidys.
Regardless of being one 12 months older (common age 7.18 years) than these handled in Half 1 (common age 5.98 years), crossover-treated sufferers confirmed clinically significant and statistically important purposeful profit for NSAA, Time to Rise (TTR) and 10-meter stroll/run (10MWR) operate checks in contrast with a pre-specified, propensity-weighted exterior management group.
In June 2024, the FDA accredited the labeled indication for Elevidys to incorporate people with Duchenne muscular dystrophy who’ve a confirmed mutation within the DMD gene and are at least 4 years previous.
Worth Motion: SRPT inventory is down 22.90% at $78.10 in the course of the premarket session on the final test Tuesday.
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