Sarepta will resume gene therapy shipments after fda review of recent patient death
Elevidys is the first gene therapy approved in the US for Duchennes muscular dystrophy–the fatal muscle-wasting disease that affects boys and young men resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients including those who can no longer walk. The FDA decision Monday significantly improves Elevidys' sales outlook in the near-term, Jefferies analyst Andrew Tsai told investors in a note after the announcement. 'The street will feel relieved about the situation suggesting meaningful stock upside potential.'
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury–a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients which remains subject to a voluntary hold following two deaths,' FDA said in its statement.
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Arab News
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