science
FDA to Reconsider Gene Therapy for Rare Childhood Brain Disorder After Earlier Rejection
The U.S. Food and Drug Administration will review an experimental gene therapy for a deadly childhood brain disorder rejected four months earlier. The move follows leadership changes at the agency. A separate trade probe targets German pharmaceutical spending plans.
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The U.S. Food and Drug Administration will reconsider approving an experimental gene therapy for a deadly and rare childhood brain disorder that it rejected four months earlier, Stat reported. The decision marks the latest in a series of apparent FDA reversals after leaders installed by the Trump administration resigned or were fired in the preceding two months.
Last week UniQure was cleared to submit an application for a Huntington’s disease gene therapy previously rejected by the agency. Former FDA commissioner Marty Makary appeared to disparage that therapy on national television. Separately the U.S.
Trade Representative launched a Section 301 investigation into a German plan to reduce spending on pharmaceutical products. The probe follows an April announcement by the German Ministry of Health of a healthcare system overhaul intended to close a $23 billion funding gap.
The original plan, which included variable discounts on pharmaceuticals, drew opposition from the German pharmaceutical industry and is being replaced.
