Vatiquinone FDA Approval Status
Last updated by Judith Stewart, BPharm on Feb 20, 2025.
FDA Approved: No
Generic name: vatiquinone
Company: PTC Therapeutics, Inc.
Treatment for: Friedreich’s Ataxia
Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase in development for the treatment of Friedreich ataxia.
- Friedreich ataxia (FA) is a rare neuromuscular disorder that is usually caused by a single genetic defect in the frataxin gene, which leads to reduced production of the mitochondrial protein frataxin. Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis. Friedreich ataxia mainly affects the central nervous system and the heart, with symptoms that include progressive loss of coordination and muscle strength, difficulty speaking, swallowing and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment.
- Vatiquinone is a small molecule, 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. It works to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing cellular inflammation and oxidative stress and promoting neuronal survival.
- A New Drug Application (NDA) has been submitted to the U.S. Food and Drug Administration (FDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia. The NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies, demonstrating significant, durable and clinically meaningful evidence of slowing disease progression on key aspects of disease.
Development timeline for vatiquinone
Further information
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