FDA investigates patient deaths after treatment with Sarepta's gene therapy
(Reuters) -The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients after receiving Sarepta Therapeutics' gene therapy, Elevidys.
In June, Sarepta reported a second death in a patient who had received its gene therapy, which raised concerns about the safety and future demand for the treatment.
The patients who died were a 16-year-old, weighing 70 kilograms, and a 15-year-old, weighing 50 kilograms. Both boys were non-ambulatory and their deaths occurred within 90 days after treatment, the company said in an investor call last week.
The two patients showed signs of acute liver failure and were hospitalized less than two months after treatment with Elevidys, the health regulator said on Tuesday. It added that it is evaluating the need for further regulatory action.
Sarepta said in an email to Reuters, "The FDA communication was triggered by our report to the FDA and our suggested update to the label to include information relating to the recent events."
The U.S. prescribing information for Elevidys warns of acute liver injury but does not mention liver failure or death.
Elevidys, approved by the FDA in 2024 for ambulatory Duchenne muscular dystrophy patients aged four and older, is the only gene therapy available for the disease. It carries a known risk of liver damage and was conditionally approved for non-ambulatory patients despite failing to meet the main goal in a late-stage study.
Sarepta said earlier this month it was investigating both cases independently and in relation to each other to identify any common risk factors.
The company has suspended its Elevidys sales forecast for 2025, halted shipments to non-ambulatory patients and is working with regulators on a new treatment plan.
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Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Evan Berland | +1 215 432 0234 | Léo Le Bourhis | +33 6 75 06 43 81 | Victor Rouault | +33 6 70 93 71 40 | Timothy Gilbert | +1 516 521 2929 | Léa Ubaldi | +33 6 30 19 66 46 | Investor RelationsThomas Kudsk Larsen | +44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Felix Lauscher | +1 908 612 7239 | Keita Browne | +1 781 249 1766 | Nathalie Pham | +33 7 85 93 30 17 | Tarik Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Yun Li | +33 6 84 00 90 72 | Sanofi forward-looking statementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans' and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. 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The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under 'Risk Factors' and 'Cautionary Statement Regarding Forward-Looking Statements' in Sanofi's annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press ReleaseSign in to access your portfolio
