• Regulatory NewsRegulatory News

    Two Gene Therapy Approvals Headline CBER’s FY 2018 Report

    The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) on Wednesday published its Fiscal Year 2018 report, highlighting two gene therapy approvals, as well as the approvals of a hepatitis B vaccine, a vaccine to prevent shingles and a flu vaccine for children as young as six months. Both of the gene therapy approvals occurred at the very beginning of the previous fiscal year, with CBER signing off on Spark Therapeutics’ Luxturna...
  • 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...