Vatiquinone for Friedreich’s ataxia
Last updated Sept. 5, 2025, by Marisa Wexler, MS
Fact-checked by Jose Lopes, PhD
What is vatiquinone for Friedreich’s ataxia?
Vatiquinone, previously known as PTC-743 and EPI-743, is an oral treatment candidate that PTC Therapeutics is developing to slow the progression of Friedreich’s ataxia (FA). The experimental therapy is designed to reduce brain inflammation and cell damage by blocking the activity of a protein called 15-lipoxygenase.
PTC had asked the U.S. Food and Drug Administration (FDA) to approve the small molecule treatment for adults and children with FA based on data from a Phase 2/3 clinical trial. However, in 2025, the FDA rejected that application, saying there was not sufficient data to prove that vatiquinone is effective as an FA treatment. The FDA said an additional study would be needed to support a resubmission of the application.
Therapy Snapshot
| Treatment Name: | Vatiquinone |
| Administration: | Taken orally three times per day |
| Clinical testing: | Phase 2/3 trial complete; open-label extension ongoing |
How will vatiquinone be administered?
Vatiquinone is available as oral capsules or as a liquid solution, both taken by mouth. In FA clinical trials, it was tested at doses of 200 mg or 400 mg, administered three times daily.
Vatiquinone in clinical trials
A Phase 2/3 trial called MOVE-FA (NCT04577352) tested vatiquinone against a placebo in 146 FA patients ages 7 and older. Most of the participants were children or adolescents.
- The main goal of MOVE-FA was to determine if vatiquinone would slow the progression of neurological symptoms as measured by the modified Friedreich Ataxia Rating Scale (mFARS) after about 1.5 years. The study failed to meet this goal; mFARS scores were overall not significantly different in patients given vatiquinone or the placebo. However, the results also showed a statistically significant slowing of upright stability decline, which PTC has argued is the most sensitive assessment in the enrolled patient population.
- Those who completed earlier studies of vatiquinone for FA had the option to join an ongoing open-label extension study (NCT05515536) in which all participants are being treated with vatiquinone and monitored for three years. An analysis that compared data from the extension study against data from a natural history study indicated that the decline of mFARS scores was about 50% slower in patients given vatiquinone.
Vatiquinone side effects
In the MOVE-FA clinical trial, adverse events reported among patients on vatiquinone included:
- headache
- diarrhea, nausea, and/or abdominal pain
- nosebleeds
- itching
- indigestion
- decreased appetite
- blood taking longer to clot
- stiffness
- chest pain
Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
FAQs about Vatiquinone
PTC Therapeutics had asked the U.S. Food and Drug Administration (FDA) to approve vatiquinone for treating people with Friedreich’s ataxia, but in 2025, the FDA rejected that application, saying available data weren’t sufficient to prove the therapy is effective. The FDA has said that an additional clinical trial would be needed to support a resubmission of the application. Therefore, it’s not clear when or if the FDA will approve vatiquinone.
Vatiquinone is an experimental therapy that has not been approved for treating Friedreich’s ataxia (FA) and is still in development. It’s designed to limit neuroinflammation and nerve cell damage in people with FA by blocking the activity of an enzyme called 15-lipoxygenase, with the ultimate goal of slowing disease progression. A Phase 2/3 clinical trial failed to show a significant effect of vatiquinone on overall neurological disability, though significant effects were seen in a subscale measuring upright stability.
Because vatiquinone is still in development, studies examining its interaction with alcohol are lacking. Patients who wish to drink alcohol while taking the medication should discuss this issue with their doctors.
In a Phase 2/3 clinical trial, vatiquinone was not better than a placebo at slowing a measure of overall neurological disability after 1.5 years of treatment, but a significant effect was seen on a measure of upright stability. More data is needed to determine if the therapy can effectively slow disability progression and, if so, how soon patients may see benefits with treatment.
Side effects of vatiquinone reported in the MOVE-FA Phase 2/3 clinical trial were headache, digestive complaints, nosebleeds, itching, blood clotting abnormalities, stiffness, and chest pain. It’s recommended that patients speak with their healthcare providers to understand how the medication may affect them. Patients should also report any unanticipated side effects of vatiquinone to their doctors.
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