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Sarepta Shares Plummet on FDA Review of Elevidys Following Third Patient Death

Regulators are weighing a voluntary pause of Elevidys shipments with a black-box warning after reports of a third fatal liver failure.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
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Sarepta Therapeutics faces renewed scrutiny as safety concerns mount around its gene therapy program.
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Overview

  • FDA Commissioner Marty Makary said he is taking a hard look at whether Elevidys should remain on the market after a third clinical trial participant died of acute liver failure.
  • The U.S. Food and Drug Administration has requested that Sarepta voluntarily halt all Elevidys shipments for now and add a black-box warning for acute liver injury and liver failure.
  • Sarepta is moving ahead with a cost-cutting plan that slashes 36% of its workforce, pauses limb-girdle gene-therapy programs and pivots R&D toward its siRNA platform to save about $400 million annually.
  • The company reported preliminary Q2 net product revenue of $513 million, including $282 million from Elevidys, and closed the quarter with approximately $850 million in cash, cash equivalents and investments.
  • Sarepta’s stock tumbled nearly 40% on Friday, reversing recent gains from its restructuring announcement as investors reacted to heightened regulatory uncertainty.