Following results from their MOVE-FA phase 3 clinical trial of the drug vatiquinone in children and adults with Friedreich’s Ataxia (FA), the pharmaceutical company PTC Therapeutics wrote to the US regulatory body the Food and Drug Administration (FDA) to determine whether the data from the study would be sufficient to support an application for New Drug Application (NDA) for accelerated approval.
The study did not meet its primary endpoint, the main measurement to show whether the drug works, as there was not a statistically significant change in the overall mFARS score (a scale that measures FA progression). However, PTC Therapeutics did observe meaningful clinical benefit in some components of mFARS, including a slowing of progression in both bulbar stability (relating to muscles in the head and neck for swallowing and coughing) and upright stability components at 72 weeks. There were also improvements in the impact of fatigue with vatiquinone treatment compared to those who were not given vatiquinone.
In their written response, the FDA stated that while they see the value of upright stability as a clinically meaningful endpoint, they believed a confirmatory study would likely be needed to support NDA submission. PTC has requested a follow-up live meeting to address the issues raised by the FDA.
PTC is participating in a scientific advice procedure with the EU regulatory body, the European Medicines Agency (EMA), to determine if the MOVE-FA data could support a conditional marketing authorisation application in the European Economic Area (EEA). PTC expects to have the outcome of this procedure in the first quarter of 2024.