First Therapy to Treat Friedreich's Ataxia Approved

March 03, 2023

In an important step forward for adolescents and adults affected by Friedreich’s ataxia (FA), on February 28th, Rare Disease Day 2023, the US Food and Drug Administration (FDA) approved Reata Pharmaceuticals’ SKYCLARYS for the treatment of FA. It becomes the first ever approved therapy for FA, representing a major milestone for the FA community.

SKYCLARYS, the oral, once-daily medication, also known as omaveloxolone, is indicated for the treatment of FA in adults and adolescents aged 16 years and older. This comes after successful clinical trials supporting the efficacy and safety of the drug in this patient population. The studies conducted ultimately demonstrated a statistically significant lower mFARS score, a clinical assessment tool used to assess severity of symptoms, in the group receiving SKYCLARYS versus the group receiving placebo.

Reata Pharmaceuticals also confirmed the company’s Marketing Authorization Application for omaveloxolone is under review in Europe by the European Medicines Agency; and that the FDA approval also granted a rare paediatric disease priority review voucher, an FDA initiative to expedite the development of drugs for rare paediatric disease populations.

Ataxia UK, the leading national charity in the UK for people affected by ataxia, have confirmed they are working and meeting with Reata Pharmaceuticals to encourage and support them in seeking UK regulatory approval for omaveloxolone.