One and Done: FDA Approves Reata’s Rare-Disease Drug Skyclarys After Single Trial

While most drug candidates must endure two recommended clinical trials to score a coveted regulatory approval, one that treats a rare, incurable neurological disorder has managed to get the U.S. Food and Drug Administration’s thumbs up from a solitary, albeit impressive, study. Reata Pharmaceuticals, a company specializing in the creation of small-molecule therapeutics, has been given the go-ahead from the FDA for its drug omaveloxolone, which has been dubbed Skyclarys for its much-anticipated market entry. It has been approved for treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. “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 the company’s Chief Executive Officer, Warren Huff. “We look forward to delivering Skyclarys to eligible patients as quickly as possible.”

Indeed, the drug is the first of its kind to pass the strict regulatory agency with only one clinical trial under its belt. Reata’s may have achieved this feat by doing a case study on the same set of patients over a period of time. This approach is more suitable for the drug’s purpose: treating a disease that affects a mere fraction of the population. In 2020, when the FDA pointed out a lack of sufficient evidence to pass on an approval, Reata couldn’t follow up quickly with another trial due to a lack of viable participants. The eventual result came from the company’s solid first trial long-term findings, which was able to demonstrate the drug’s ability to improve neurological functions compared to a placebo.

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