PTC puts Friedreich ataxia drug's past failures in the rearview with long-term data drop

Several months after forging a fresh path forward for its troubled Friedreich ataxia (FA) drug vatiquinone, PTC Therapeutics is back with some much-needed positive data.

The candidate passed muster by hitting pre-specified endpoints in two separate long-term extension studies, the company said Tuesday, with a “highly statistically significant” and durable effect on disease progression.