FDA grants priority review to vatiquinone application for FA
PTC Therapeutics says it expects decision by August
The U.S. Food and Drug Administration (FDA) granted priority review to PTC Therapeutics‘ application seeking the approval of vatiquinone, an oral treatment for children and adults with Friedreich’s ataxia (FA).
The agency will announce a decision by Aug. 19, the company said.
“We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich’s ataxia,” Matthew B. Klein, MD, CEO of PTC, said in a company press release. “If approved, vatiquinone would be the first therapy for pediatric patients with FA, and provide a potential safe, well-tolerated and effective treatment alternative for adults.”
Vatiquinone (PTC-743) is a small molecule designed to block the activity of 15-lipoxygenase, an enzyme that regulates neuroinflammation processes involved in FA.
Positive results from the now-complete Phase 2/3 MOVE-FA clinical trial (NCT04577352) supported PTC’s application. The trial tested the therapy in 146 FA patients, ages 7 and older, who could walk at least 10 feet in one minute, with or without assistance. Participants were randomly assigned vatiquinone, taken three times daily, at a weight-based dose, or a placebo over 72 weeks, or about 1.5 years.
Benefits for children with FA
Data showed the therapy failed to outperform the placebo in slowing disease progression, as indicated by the modified Friedreich Ataxia Rating Scale, or mFARS. Despite these results, vatiquinone had a statistically significant effect on the mFARS upright stability subscale, which, according to PTC, is “now understood to be the most sensitive and relevant for the enrolled primary analysis population.”
Better upright stability underscored favorable benefits in functionality, as assessed by the 1-minute walk distance test and the functional component of the Modified Fatigue Rating Scale.
These benefits are noteworthy for pediatric patients, who comprised the majority of MOVE-FA participants. Skyclarys (omaveloxolone), the sole FA treatment to gain FDA approval, is only authorized for those ages 16 and older.
“The granting of priority review by FDA reflects the significant unmet need for younger patients with FA,” Klein said.
Upon completion, MOVE-FA participants could join an extension study in which all were given long-term vatiquinone therapy. Almost three years of treatment showed that vatiquinone slowed mFARS score decline by about 50% compared with untreated patients taking part in a large natural history study known as FACOMS.
Late last year, a study led by PTC scientists confirmed that vatiquinone didn’t increase the risk of heart problems when tested in healthy adults, including at doses about 3.5 times higher than the therapeutic dose.
“We look forward to working collaboratively with FDA during the review process,” Klein said.
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