On 19th August 2025, the pharmaceutical company PTC Therapeutics announced the disappointing news that the US drug regulators the FDA have not approved the drug Vatiquinone to treat adults and children with Friedreich’s ataxia (FA). The FDA has said that substantial evidence of the drug’s effectiveness was not demonstrated, and an additional clinical trial is needed.
PTC plans to meet with the FDA to discuss next steps. We will share any updates with the community through our channels.
Read PTC’s letter to the community here.
Read more about Vatiquinone here.
