The FDA has approved Skyclarys, Reata Pharmaceuticals’ oral, once-daily medication to treat Friedreich’s ataxia in people aged 16 years and older.

According to a release from Reata, Skyclarys (omaveloxolone) previously received orphan drug, fast track and rare pediatric disease designations from the FDA. With this approval, the agency granted a rare pediatric disease priority review voucher.

Announcement of the drug’s approval came 4 months after the FDA stated it would not hold an advisory committee meeting over Reata’s NDA.

“The approval of Skyclarys, the first therapy specifically indicated for the treatment of Friedreich’s ataxia, is an important milestone for patients affected by this disease as well as their families and caregivers,” Reata CEO Warren Huff stated in the release. “We are grateful to Friedreich’s ataxia patients, investigators, U.S. regulators and our scientists and employees who made this approval possible.”

Approval of Skyclarys was bolstered by positive safety and efficacy data from the MOXIe part 2 trial as well as a post-hoc propensity-matched analysis of the open-label extension trial, according to the release.

The randomized, double-blind, placebo-controlled, 48-week MOXIe part 2 trial included patients with Friedreich’s ataxia and baseline modified Friedreich’s Ataxia Rating Scale (mFARS) scores between 20 and 80, who were given either 150 mg of Skyclarys or placebo daily. The study’s primary endpoint was change from baseline in mFARS scores.

According to the release, treatment with Skyclarys resulted in statistically significant lower mFARS scores, or reduced impairment, compared with placebo at 48 weeks. The most common adverse effects reported were elevated liver enzymes, headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain.

In the post-hoc analysis, patients treated with 150 mg Skyclarys in the MOXIe open-label extension study had lower mFARS scores after 3 years compared with matched, untreated patients.

“Today’s approval of Skyclarys represents a significant milestone in our effort to advance research and achieve treatments for Friedreich’s ataxia,” Jen Farmer, Friedreich’s Ataxia Research Alliance CEO, said in the release. “The entire Friedreich’s ataxia community, including patients, clinicians, scientists, pharmaceutical companies, government agencies and others, have worked collaboratively for decades to enable therapeutic development for this debilitating disease.”