On 8th October 2024, the pharmaceutical company PTC Therapeutics announced positive results from their phase III trial of their drug Vatiquinone for Friedreich’s ataxia (FA).
Analysis of their MOVE-FA long-term study demonstrated that 144 weeks of Vatiquinone treatment resulted in a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale (mFARS) relative to untreated individuals with FA from a natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.
This treatment difference represents a 50% slowing in disease progression over 3 years. PTC plan to submit a new drug application to the FDA in the US in December 2024. Read their press release here.
You can read our previous article on the trial here.
