其 他 安 全 警 示
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| The United States: FDA recommends removal of voluntary hold for Elevidys for ambulatory patients (English only) |
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The United States Food and Drug Administration (FDA) is recommending the removal of the voluntary hold for ambulatory patients who may now receive Elevidys (delandistrogene moxeparvovec-rokl), a Sarepta Therapeutics gene therapy for Duchenne Muscular Dystrophy (DMD). The FDA’s investigation has concluded that the death of the 8-year-old boy is unrelated to the gene therapy product itself.
The FDA will continue to work with the sponsor regarding non ambulatory patients, which remains subject to a voluntary hold, following two deaths.
The patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD.
Elevidys is an adeno-associated virus vector-based gene therapy using Sarepta Therapeutics, Inc.’s AAVrh74 Platform Technology for the treatment of Duchenne muscular dystrophy. The product is administered as a single intravenous dose. Duchenne muscular dystrophy is a rare genetic condition characterized by progressive muscular weakness. The disease occurs due to a defective gene.
Please refer to the following website in FDA for details:
http://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients
In Hong Kong, the above product is not a registered pharmaceutical product. Related news was previously issued by Singapore Health Sciences Authority and FDA, and was posted on the Drug Office website since 25 Mar 2025, with the latest update posted on 26 Jul 2025.
Ends/Tuesday, Jul 29, 2025
Issued at HKT 17:00
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