Reata Pharmaceuticals gets Skyclarys FDA approval for Friedreich’s Ataxia

Reata Pharmaceuticals has secured the approval of the US Food and Drug Administration (FDA) for Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia, a neuromuscular disease.

Skyclarys is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in the US.

Patients are recommended to take Skyclarys capsules orally without food once a day at a dosage of 150mg, said the Nasdaq-listed biopharma company.

Skyclarys FDA approval was backed by the efficacy and safety results from the MOXIe part 2 trial and a post hoc propensity-matched analysis of the open-label MOXIe Extension trial.

Warren Huff — Reata Pharmaceuticals CEO, commenting on Skyclarys FDA approval, said: “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.

“As a company, this is a transformative milestone that highlights our commitment to developing and commercializing novel therapies for patients with severe diseases with few or no approved therapies.

“We look forward to delivering Skyclarys to eligible patients as quickly as possible.”

The MOXIe part 2 study enrolled 103 patients with Friedreich’s ataxia who received placebo or Skyclarys 150mg for 48 weeks.

Patients receiving Skyclarys performed better on the modified Friedreich’s Ataxia Rating Scale (mFARS) score compared to those receiving placebo.

In post hoc analysis, patients who continued treatment with Skyclarys in an open-label extension for up to three years performed better on the mFARS than a matched set of untreated patients from a natural history study.

Reata Pharmaceuticals said the commercial drug supply of Skyclarys will be available in Q2 2023 in the US.

The marketing authorization application for Skyclarys is under review in Europe by the European Medicines Agency.

Skyclarys had earlier received orphan drug, fast track, priority review, and rare pediatric disease designations.

Reata Pharmaceuticals is also developing bardoxolone methyl for chronic kidney disease treatment and cemdomespib for diabetic neuropathic pain.