PTC Therapeutics Inc. has submitted the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA)—marking the fourth approval application PTC has submitted to the FDA this year.
 
“We are excited to have reached this important milestone in the development of vatiquinone,” said Matthew B. Klein, M.D., CEO of PTC Therapeutics. “The evidence of short- and long-term efficacy as well as the extensive safety data collected, particularly in children, supports the potential for vatiquinone to fill the significant unmet need for children living with Friedreich ataxia as well as provide a potential treatment option for adults living with FA.”
 
The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. 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 demonstrate that vatiquinone is safe and well-tolerated in all age groups studied.