• Regulatory NewsRegulatory News

    Sarepta Wins Controversial FDA Approval for First DMD Drug

    The US Food and Drug Administration (FDA) on Monday approved Sarepta Therapeutics’ first drug to treat patients with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness in young children. The approval is highly controversial after a FDA advisory committee voted against approval in April as the outside experts said there was not substantial evidence that the drug is effective in providing clinical benefit...
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    Republican Senators Lobby FDA Ahead of Decision on Sarepta’s DMD Drug

    The contentious debate over whether the US Food and Drug Administration (FDA) will approve or deny Sarepta’s Duchenne Muscular Dystrophy (DMD) drug eteplirsen will come to an end either on or before Thursday of this week. But before that decision is made, two Republican senators, Ron Johnson (WI), Chairman of the Committee on Homeland Security and Governmental Affairs, and Dan Coats (IN), sent a letter to FDA Commissioner Robert Califf last Friday to “express disapp...
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    FDA Panel Votes Against Approval for Sarepta DMD Drug

    Following a contentious debate pitting young boys with the terminal illness Duchene Muscular Dystrophy (DMD) against the US Food and Drug Administration (FDA), an advisory panel of outside experts on Monday voted against the approval of Sarepta Therapeutics’ controversial investigational drug eteplirsen to treat DMD. The agency’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7-3 against approval, with three abstaining, and 7-6 against the accelerat...
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    Academic DMD Experts Criticize ‘Errors’ in FDA Ad Com Briefing Documents

    More than 35 medical professors and experts have criticized the US Food and Drug Administration’s (FDA) review of a Duchenne Muscular Dystrophy (DMD) drug, citing errors in the agency’s advisory committee meeting briefing documents. At issue is Sarepta’s drug eteplirsen, which the company is seeking approval as the world’s first treatment for DMD, a rare and fatal genetic disorder afflicting about 15,000 boys in the US. The drug was initially supposed to go before the Pe...