NasdaqGS:PTCTBiotechs
FDA Request for More Data on Vatiquinone Might Change the Case for Investing in PTC Therapeutics (PTCT)
On August 19, 2025, PTC Therapeutics announced that the U.S. Food and Drug Administration issued a Complete Response Letter for the New Drug Application of vatiquinone for Friedreich's ataxia, stating substantial evidence of efficacy was not shown and that an additional controlled study is needed for approval.
This setback highlights the regulatory hurdles biopharmaceutical companies face in bringing rare disease therapies to market, even after multiple clinical studies and years of...