• ExplainersExplainers
    Regulatory NewsRegulatory News

    Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers

    Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers (PRVs) which allow its recipient to expedite the review of any one of its new drug products. What are these vouchers, why is FDA issuing them and what benefit might they have for society? Find out in our latest Regulatory Explainer on the Priority Review Voucher system. Last updated on 25 February 2020 to include: Vifor Pharma said on 17 February that it p...
  • 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

    FDA Approves New Gene Therapy Targeting Specific Mutation

    The US Food and Drug Administration (FDA) on Tuesday approved a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness. The approval comes two months and a week after an advisory committee of outside experts unanimously voted in favor of approving the treatment for the condition which affects about 6,000 worldwide. The price of the new gene therapy, which Leerink...
  • Regulatory NewsRegulatory News

    FDA Panel Votes Unanimously in Favor of Gene Therapy to Treat Rare Eye Disorder

    Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 t...
  • Regulatory NewsRegulatory News

    Regulating CRISPR: FDA and Industry Offer Perspective

    Although the US market is likely more than a year or two away from seeing any commercialized medical products that rely on CRISPR-Cas9 technology, the rapidly developing field has already grabbed the attention of the US Food and Drug Administration (FDA) and other drug regulators. What is CRISPR? According to the Broad Institute , CRISPR (pronounced "crisper") stands for Clustered Regularly Interspaced Short Palindromic Repeats, which form the basis for a genome ...
  • Regulatory NewsRegulatory News

    EU Court of Justice Denies EMA Appeal to Release Documents on Two Medicines

    The Court of Justice of the EU earlier this month delivered a blow to transparency efforts at the European Medicines Agency (EMA), dismissing two appeals by the regulator. The decision means the court has upheld the suspension of the release of clinical study report documents requested by a pharmaceutical company related to PTC Therapeutics’ Duchenne muscular dystrophy treatment Translarna (ataluren) and three toxicity studies for Intervet’s Bravecto (fluralaner), a vete...
  • 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

    BIO Chair: Trump Should Re-Appoint Califf to Head FDA

    In a phone interview with Focus on Thursday, Ron Cohen, MD, president and CEO of Acorda Therapeutics and chair of industry group BIO, offered his views on increasing funding for the US National Institutes of Health (NIH), what the future may hold for the US Food and Drug Administration (FDA), as well as some new ideas and possible solutions to help contain rising drug prices. NIH and FDA For NIH, Cohen echoed sentiments circulating on Capitol Hill around the 21s...
  • 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

    EMA Contests Two Judicial Decisions Over Clinical Trial Transparency Efforts

    The European Medicines Agency (EMA) on Thursday announced it has appealed two interim orders by the President of the General Court of the EU, which suspended the release of clinical study documents requested by third parties under a new transparency regulation. The first order blocked the release of a study report for PTC Therapeutics’ Translarna (ataluren), a Duchenne’s muscular dystrophy treatment, until a final ruling is provided. EMA says it was planning to provid...
  • 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...