Other safety alerts
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| The United States: FDA investigating deaths due to acute liver failure in non-ambulatory Duchenne Muscular Dystrophy patients following ELEVIDYS |
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The United States Food and Drug Administration (FDA) announces that since approval, it has received two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male patients with Duchenne Muscular Dystrophy (DMD) with ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy.
The two reported deaths in non-ambulatory DMD patients appear to be related to treatment with ELEVIDYS. Reports were received from clinical trial and postmarketing data. In these two cases, the patients presented with elevated transaminases and were hospitalized less than two months after treatment with ELEVIDYS.
FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following ELEVIDYS, and is evaluating the need for further regulatory action.
The U.S. prescribing information (USPI) includes information on the risk of acute serious liver injury following treatment with ELEVIDYS under Warnings and Precautions, Adverse Reactions and Patient Counseling Information, but does not include warnings regarding liver failure or death.
Please refer to the following website in FDA for details:
http://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients
In Hong Kong, the above product is not a registered pharmaceutical product. Related news was previously issued by Singapore Health Sciences Authority, and was posted on the Drug Office website on 25 Mar 2025 and 24 Jun 2025.
Ends/Wednesday, Jun 25, 2025
Issued at HKT 15:45
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