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.
Update 19th February 2026
On February 19th 2026, PTC Therapeutics provided an update via press release. This update explained that in December 2025, as planned, they had a meeting with the FDA to discuss next steps. The FDA indicated to PTC Therapeutics that an additional study is needed to support them resubmitting an NDA (New Drug Application) for vatiquinone, and that this could be an open-label study with a natural history control group. To read the press release click here.
