Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy - Seite 2
The conditional and time-limited approval pathway in Japan provides for marketing authorization in Japan for up to seven years for innovative medicines to treat serious conditions. To be eligible for the pathway, certain additional criteria must also be met, including early clinical trial results that have demonstrated significant efficacy and safety.
As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche.
About ELEVIDYS (delandistrogene moxeparvovec-rokl)
ELEVIDYS (delandistrogene moxeparvovec-rokl) 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.
ELEVIDYS is indicated in U.S. for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.
- For patients who are ambulatory and have a confirmed mutation in the DMD gene
- For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.
The DMD indication in non-ambulatory patients is approved under accelerated approval in the U.S. based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
U.S. IMPORTANT SAFETY INFORMATION
CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
WARNINGS AND PRECAUTIONS:
Infusion-related Reactions:
- Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
- ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
- Discontinue infusion for anaphylaxis.
Acute Serious Liver Injury: