The US Food and Drug Administration (FDA) has approved 鈥� oral, once-daily medication patients.
SKYCLARYS is indicated to treat the ultra-rare, inherited neurodegenerative disorder in 16 years and above-aged adults and adolescents in the US. SKYCLARYS is the first approved treatment in Friedrich鈥檚 Ataxia and marks a milestone for drug development in this complex disease. There are .
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By GlobalDataThe FDA granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations for the medication.
The European Medicines Agency (EMA) is also reviewing Reata鈥檚 marketing authorisation application (MAA) for SKYCLARYS in Europe.
It also received Orphan Drug designation from the European Commission for treating the disorder.
CEO Warren Huff said: 鈥淭he approval of SKYCLARYS, the first therapy specifically indicated for the treatment of , is an important milestone for patients affected by this disease, as well as their families and caregivers.
鈥淲e are grateful to patients, investigators, US regulators, and our scientists and employees who made this approval possible.
鈥淎s a company, this is a transformative milestone highlighting our commitment to developing and commercialising novel therapies for patients with severe diseases with few or no approved therapies.鈥�
The FDA approved SKYCLARYS based on the safety and efficacy data obtained from the MOXIe Part 2 randomised, double-blind, placebo-controlled trial and a post hoc Propensity-Matched 网曝门 of the open-label MOXIe Extension trial.
In the MOXIe Part 2 study, patients with genetically confirmed Friedreich鈥檚 ataxia and baseline-modified Friedreich鈥檚 Ataxia Rating Scale (mFARS) scores between 20 and 80 were randomised in a 1:1 ratio and given placebo or SKYCLARYS 150mg daily.
The trial鈥檚 primary endpoint was a change from baseline in mFARS score against placebo at Week 48 in the Full 网曝门 Population of patients without severe pes cavus.
