PTC Therapeutics has submitted vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the US Food and Drug Administration (FDA).

"We are excited to have reached this important milestone in the development of vatiquinone. 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," said Matthew B. Klein, MD, Chief Executive Officer of PTC Therapeutics.

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.

The vatiquinone NDA is the fourth approval application PTC has submitted to the FDA this year. The BLA for its AADC gene therapy was submitted in March 2024 and approved in November 2024. NDAs for sepiapterin, for the treatment of adults and children living with phenylketonuria (PKU) and for Translarna™ (ataluren) for the treatment of boys and young men with nonsense mutation Duchenne muscular dystrophy, have been also submitted and accepted for review.

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia.

Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing cellular inflammation and oxidative stress and promoting neuronal survival.

Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms.