FDA approves omaveloxolone for pediatric use

The FDA has approved omaveloxolone (SKYCLARYS, Reata Pharmaceuticals) for use in adult and pediatric patients aged 16 years and older with Friedreich’s ataxia, granting the medication a rare pediatric disease priority review voucher.

Friedreich’s ataxia is a debilitating condition that can lead to a shorter lifespan. A trinucleotide repeat expansion occurs in the first intron of the frataxin gene, responsible for encoding the mitochondrial protein frataxin. This can cause impaired transcription and reduced frataxin expression, leading to poor cellular iron regulation because of mitochondrial iron overload.

Symptoms of Friedreich’s ataxia include muscle weakness, loss of coordination, and fatigue. Patients with Friedreich’s ataxia will often need a wheelchair in their 20s. Data from insurance claims indicates about 5000 patients are afflicted with Friedreich’s ataxia in the United States.

“Friedreich's ataxia is a debilitating neuromuscular disease that progressively robs patients of their mobility and independence,” said Susan Perlman, MD, clinical professor, department of neurology, 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,” Perlman added.

Omaveloxolone is a medication taken orally once per day. It has been given Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA. The most recent approval was based on safety and efficacy data from the MOXIe phase 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial.

In the MOXIe phase 2 trial, patients confirmed to have Friedreich's ataxia were randomized into a placebo or omaveloxolone group. Those in the latter group received 150 mg omaveloxolone daily. The primary endpoint was a change in modified Friedreich’s Ataxia Rating Scale (mFARS) score at week 48.

Those in the omaveloxolone group saw a significant reduction in mFARS at week 48 compared to placebo. This indicates efficacy in treating Friedreich's ataxia.

Reference

Reata Pharmaceuticals announces FDA approval of SKYCLARYS (omaveloxolone), the first and only drug indicated for patients with Friedreich’s ataxia. Reata. February 28, 2023. March 2, 2023. https://www.reatapharma.com/investors/news/news-details/2023/Reata-Pharmaceuticals-Announces-FDA-Approval-of-SKYCLARYS-Omaveloxolone-the-First-and-Only-Drug-Indicated-for-Patients-with-Friedreichs-Ataxia/default.aspx