Vaquitinone News
- Márcio
- Feb 19
- 1 min read
Important day for the Fredericos!
Today, February 19, 2025, pharmaceutical company PTC Therapeutics announced that the FDA accepted priority review of the application for approval of the drug Vatiquinone for the treatment of children and adults with Friedreich's Ataxia (FA).
Vatiquinone is a small molecule, a selective 15-lipoxygenase (15-LO) inhibitor. Inhibiting 15-LO helps mitigate the consequences of mitochondrial dysfunction and oxidative stress that occur in Friedreich's ataxia, thereby reducing cellular inflammation and oxidative damage and promoting neuronal survival. Although it is not a cure for FA, vaquitinone has been evaluated in several clinical studies, many of them focused on pediatric patients, and has demonstrated good results in reducing the risk of mortality and improving symptoms.
If Vaquitinone is approved by the FDA, it will be the second drug approved by the agency capable of modifying the course of Friedreich's Ataxia (the first, already approved by the FDA and currently under analysis by Anvisa in Brazil, is Skyclarys).
