On 8th October 2024, the pharmaceutical company PTC Therapeutics announced positive results from their phase III trial of their drug Vatiquinone for adults and children with 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.
Update from 19/12/2024
On 19th December 2024, PTC Therapeutics announced they have submitted a New Drug Application to the FDA (the US drug regulators), for vatiquinone as a treatment for FA in children and adults.
This submission is based on data from the MOVE-FA study and two long term studies in children and adults with FA.
Read their press release here.
