Biopharmaceutical company PTC Therapeutics Inc (NASDAQ: PTCT) announced on Thursday that it has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia (FA), a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.

The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study, as well as results from two long-term studies including paediatric and adult FA patients. PTC said that data from these three studies demonstrate significant, durable and clinically meaningful evidence of slowing disease progression on key aspects of disease. In addition, these studies show that vatiquinone is safe and well tolerated in all age groups studied.

This is the fourth approval application PTC has submitted to the FDA this year.