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  • Regulatory NewsRegulatory News

    Time for FDA to Release Complete Responses Letters? Debate Reignites

    The idea of the US Food and Drug Administration (FDA) releasing complete response letters (CRLs) resurfaced this week as Sarepta Therapeutics received a CRL but refused to make public any portion of it. Following a question on transparency at an event Thursday , Sarepta CEO Doug Ingram said that not releasing the text of the CRL was “a service to the community” and that FDA might not want a confidential letter to be released. “The basis for the CRL is exactly as I've s...
  • Regulatory NewsRegulatory News

    Sarepta Sells Priority Review Voucher for $125M to Gilead

    Sarpeta Therapeutics on Tuesday sold its priority review voucher (PRV) to Gilead for $125 million, speeding up the US Food and Drug Administration (FDA) approval process for any future drug or biologic of Gilead’s choosing from 10 months to six months. The voucher was awarded to Sarepta under the rare pediatric PRV program after winning FDA approval for its Duchenne Muscular Dystrophy (DMD) drug Exondys 51 (eteplirsen) in September 2016. This is the third PRV Gilead ...
  • Regulatory NewsRegulatory News

    FDA and Industry: How Dangerous is the Revolving Door?

    Earlier this week, a former deputy director of the US Food and Drug Administration’s (FDA) Office of Generic Drugs (OGD) settled charges that he provided tips on drug approvals to three hedge fund managers that made tens of millions off the non-public information. The settlement raises some important questions on the so-called revolving door between industry and FDA, and what it means for patient safety. Is it OK for those in the pharmaceutical and medical device indust...
  • Regulatory NewsRegulatory News

    Pediatric Priority Review Vouchers on the Chopping Block as Reauthorization Stalls

    Congress and President Barack Obama have one week to reauthorize the rare pediatric priority review voucher (PRV) program or else it will end on 30 September 2016, after seven pediatric PRVs have been awarded, one of which sold for $350 million last year. The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments, affecting fewer than 200,000 patients under the age of 18. The vouchers can be used to speed FDA’s review ...
  • 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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    FDA Faces Sensitive Challenge With Review of DMD Drug

    The blanketing of snow on the East Coast this past weekend prompted the postponement of a high-profile US Food and Drug Administration (FDA) advisory committee hearing for a Duchene Muscular Dystrophy (DMD) drug that is stirring up public controversy and will eventually lead to an up or down decision testing FDA’s resilience to public pressure. The advisory committee meeting, which the Jett Foundation, a DMD advocacy group, said Tuesday will likely be rescheduled withi...
  • ReconRecon
    Regulatory NewsRegulatory News

    Regulatory Recon: Shkreli Arrested on Securities Fraud Charges (17 December 2015)

    • 17 December 2015
    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher , @Zachary Brennan and @RAPSorg on Twitter. In Focus: US Shkreli, CEO Reviled for Drug Price Gouging, Arrested on Securities Fraud Charges ( Indictment )( Bloomberg ) ( New York Times ) ( Reuters ) FDA Schedules Advisory Committee to Review Sarepta DMD Drug ( FDA ) HHS OIG Report on...