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Delandistrogene moxeparvovec
Gene therapy medication From Wikipedia, the free encyclopedia
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Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy.[3] It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells.[3] It is an adeno-associated virus vector-based gene therapy that is given by intravenous infusion (slow injection into a vein).[1]
The most commonly reported side effects include vomiting, nausea, liver failure, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood).[3] Three patients may have died from acute liver failure.[4][5][6] Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023.[3][7] It was developed by Sarepta Therapeutics, together with Roche, and is manufactured by Catalent.[8]
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Medical uses
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Perspective
Delandistrogene moxeparvovec is indicated for the treatment of ambulatory and non-ambulatory individuals four years of age and older with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.[1][2][3][9]
Delandistrogene moxeparvovec is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. FDA states that the conditional approval is based on detection of successful gene expression; evidence of clinical improvement is still pending.[3]
In the phase 3 randomized clinical trial published in Nature Medicine, Elevidys AAV gene therapy for Duchenne muscular dystrophy failed to show statistically significant motor function improvement. The study included 125 ambulatory male patients aged 4-8 years (63 in therapy group, 62 in placebo group). Mean motor function assessment score changes were 2.57 points in the Elevidys group and 1.92 points in the placebo group, with a non-significant difference of 0.65 points at the 52-week endpoint.[10]
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History
The accelerated US Food and Drug Administration (FDA) approval of delandistrogene moxeparvovec was based on data from a randomized clinical trial that established that delandistrogene moxeparvovec increased the expression of the designed micro-dystrophin protein in delandistrogene moxeparvovec-treated individuals aged four to five years with Duchenne muscular dystrophy.[3] On 18 July 2025, the FDA announced that it had asked Sarepta Therapeutics to suspend distribution of Elevidys and to pause clinical trials on other gene therapy products still in development, following the recent deaths of three patients that had received either Elevidys or an experimental gene therapy using the same viral platform. In all three cases, the cause of death appeared to be acute liver failure. Sarepta has continued to supply the medication to patients despite the concerns raised at the FDA.[11]
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Pricing
Initial pricing was announced at US$3.2 million for a single treatment which is expected to last a lifetime.[12]
References
External links
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