The Plano, Texas-based Reata Pharmaceuticals has won FDA approval for Skyclarys (omaveloxolone), the first therapy specifically indicated to treat Friedreich’s ataxia. The drug is approved for use in adults and adolescents aged 16 years and older. The approval also comes with a rare pediatric disease priority review voucher.
Friedreich’s ataxia is a genetic disease that affects the nervous system, causing a gradual decline in coordination, muscle strength, and energy. This can lead to difficulty with movement and reliance on a wheelchair for people in their teens or early twenties. About 5,000 people in the U.S. are diagnosed with this condition annually.
A silencing of frataxin, a protein encoded by the FXN gene, is responsible for the disease.
$370,000 launch priceReata announced that Skyclarys would have a wholesale acquisition price of $370,000 annually.
In a company presentation, the company said not Friedreich’s ataxia pat…