Reata receives FDA OK for Skyclarys

Reata Pharmaceuticals has received permission from the Food and Drug Administration for Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.

"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," said Warren Huff, Reata's CEO. "We are grateful to Friedreich’s ataxia patients, investigators, U.S. regulators and our scientists and employees who made this approval possible. 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."

Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder typically diagnosed during adolescence—patients with Friedreich’s ataxia experience progressive loss of coordination, muscle weakness and fatigue. The condition commonly progresses to motor incapacitation and wheelchair reliance by their teens or early twenties, and eventually death. Friedreich’s ataxia affects approximately 5,000 diagnosed patients in the United States. 

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“Friedreich's ataxia is a debilitating neuromuscular disease that progressively robs patients of their mobility and independence,” said Susan Perlman, clinical professor of the department of neurology at the David Geffen School of Medicine, UCLA. “The approval of Skyclarys represents an important step forward in the treatment of Friedreich's ataxia, providing physicians with the first disease-specific treatment option approved for patients living with this ultra-rare and progressive disease.”

"Today’s approval of Skyclarys represents a significant milestone in our effort to advance research and achieve treatments for Friedreich’s ataxia," said Jen Farmer, CEO at Friedreich's Ataxia Research Alliance. "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. Today, we celebrate the impact of an engaged patient community, and we are grateful to the FDA and Reata for working together on the approval of Skyclarys, the first therapy approved in the United States for adult and adolescent patients aged 16 years and older with Friedreich's ataxia.”

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The company expects Skyclarys to be available in the second quarter of 2023.