2023-03-02| Trials & Approvals

Reata’s Groundbreaking Therapy for an Ultra-rare Neuromuscular Disease Secures FDA Approval

by Richard Chau
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Reata Pharmaceuticals, a Texas-based pharmaceutical company, recently announced that the company’s SKYCLARYS (omaveloxolone) has received approval from the U.S. FDA for the treatment of Friedreich’s ataxia (FRDA) in adults and adolescents aged 16 years and older. 

The approval of SKYCLARYS makes it the first (and by far the only) disease-specific therapy approved in the United States for patients suffering from Friedreich’s ataxia, a progressive and debilitating neuromuscular disorder. Along with this approval, the FDA has also granted Reata a rare pediatric disease priority review voucher. 

Related article: Big Pharma and Non-Profits Join NIH, FDA in New Rare Diseases Drive

An Ultra-rare Inherited Disease Damaging the Nervous System

FRDA is an incurable degenerative genetic disease that progressively damages the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, a loss of sensation in the arms and legs, poor balance, difficulty walking, as well as impaired speech and swallowing. 

Life-threatening heart problems and diabetes may also develop in patients as the disease advances, leading to a shortened lifespan. It is estimated that the disease affects about one in every 50,000 people in the U.S. and there are approximately 5,000 diagnosed patients across the country.

Success of Reata in Phase 2 Trial Supports the FDA Approval 

Reata’s SKYCLARYS is indicated for the treatment of FRDA in adults and adolescents over 16 years of age which is administered as a once-daily oral medication. In May 2022, the drug was already granted the Rare Pediatric Disease Designation and Priority Review Designation by the FDA. 

The efficacy and safety of Skyclarys to treat Friedreich’s ataxia were evaluated in the MOXIe Part 2 trial, a 48-week Phase 2 study, and the open-label MOXIe Extension trial. Efficacy data showed that participants receiving SKYCLARYS performed better on the modified Friedreich’s Ataxia Rating Scale (mFARS) than those receiving a placebo, meaning that the drug was effective in slowing down the disease progression in FRDA patients. In terms of safety, it was found that the drug was generally safe and well-tolerated. The overall positive efficacy and safety data provided the basis for the FDA approval.

Warren Huff, Chairman and Chief Executive Officer of Reata, expressed his delight with the FDA’s decision, pointing out that the approval of SKYCLARYS is an important milestone for FRDA patients as well as their families and caregivers, and he is eager to see the product commercialized and available to eligible patients in the near future.

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