Delandistrogene moxeparvovec
| Clinical data | |
|---|---|
| Trade names | Elevidys |
| Other names | SRP-9001, delandistrogene moxeparvovec-rokl |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a623058 |
| License data | |
| Routes of administration | Intravenous infusion |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
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]
Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023.[3][4] It was developed by Sarepta Therapeutics, together with Roche, and is manufactured by Catalent (a subsidiary of Novo Nordisk).[5]
Medical uses
[edit]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][6]
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 III randomized clinical trial, delandistrogene moxeparvovec failed to show statistically significant motor function improvement. The study included 125 ambulatory males aged 4-8 years (63 in therapy group, 62 in placebo group).[7]
History
[edit]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]
Society and culture
[edit]Legal status
[edit]In July 2025, the US Food and Drug Administration placed Elevidys on clinical hold;[8] and requested that Sarepta Therapeutics suspend distribution of Elevidys and pause clinical trials on other gene therapy products still in development, following the deaths of three people that had received either Elevidys or an experimental gene therapy using the same viral platform.[9] In the three cases, the cause of death appeared to be acute liver failure.[9] For a short time, Sarepta continued to supply the medication despite the concerns raised by the FDA.[9] On July 21st 2025, a few days after the initial announcement, the company placed a voluntary hold on the medication in accordance with the wishes of the FDA.[10] On July 28th 2025, the clinical hold on Elevidys was lifted after the FDA recommended the company remove the voluntary pause.[11]
In July 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency recommended the refusal of a marketing authorization for Elevidys, a medicine intended for the treatment of Duchenne muscular dystrophy.[12]
Economics
[edit]Initial pricing is US$3.2 million for a single treatment which is expected to last a lifetime.[13]
References
[edit]- ^ a b c "Elevidys- delandistrogene moxeparvovec-rokl kit". DailyMed. 22 June 2023. Archived from the original on 29 August 2023. Retrieved 29 August 2023.
- ^ a b "Elevidys". U.S. Food and Drug Administration (FDA). 22 June 2023. Archived from the original on 5 July 2023. Retrieved 22 June 2023.
- ^ a b c d e f g "FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy" (Press release). U.S. Food and Drug Administration (FDA). 22 June 2023. Archived from the original on 29 November 2023. Retrieved 22 June 2023.
This article incorporates text from this source, which is in the public domain.
- ^ "Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy" (Press release). Sarepta Therapeutics. 22 June 2023. Archived from the original on 23 June 2023. Retrieved 22 June 2023 – via Business Wire.
- ^ "Catalent inks deal to manufacture Sarepta's DMD gene therapy". 5 January 2023. Archived from the original on 17 February 2023. Retrieved 23 June 2023.
- ^ "FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy". U.S. Food and Drug Administration (FDA). 20 June 2024. Archived from the original on 21 June 2024. Retrieved 21 June 2024. This article incorporates text from this source, which is in the public domain.
- ^ Jackson J. "$3.2 million per dose Elevidys fails to meet primary endpoint in phase 3 trial". Medical Xpress. Retrieved 25 November 2024.
- ^ "FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths". U.S. Food and Drug Administration (FDA) (Press release). 18 July 2025. Retrieved 27 July 2025. This article incorporates text from this source, which is in the public domain.
- ^ a b c Kansteiner F (21 July 2025). "Sarepta stands behind Elevidys after FDA requests gene therapy be pulled from market". Fierce Pharma. Retrieved 24 July 2025.
- ^ "Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the U.S." Sarepta Therapeutics. 21 July 2025.
- ^ "FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy". Sarepta Therapeutics. 28 July 2025.
- ^ "Elevidys EPAR". European Medicines Agency (EMA). 25 July 2025. Retrieved 27 July 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ Saltzman J, Weisman R (22 June 2023). "Cambridge biotech Sarepta wins fast-track approval for the first muscular dystrophy gene therapy". The Boston Globe. Archived from the original on 5 July 2023. Retrieved 23 June 2023.
External links
[edit]- Clinical trial number NCT04626674 for "A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)" at ClinicalTrials.gov
