The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for omaveloxolone for the treatment of Friedreich ataxia.
Friedreich ataxia is a rare genetic disease caused by mutations in the FXN gene. This leads to a deficiency of frataxin, which results in mitochondrial iron accumulation and oxidative stress. The disease is typically characterized by a progressive decline in coordination, muscle weakness, and fatigue that commonly results in motor incapacitation.
Omaveloxolone is an investigational Nrf2 activator that works by improving mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The submission is supported by data from the MOXIe trial (ClinicalTrials.gov Identifier: NCT02255435), a 2-part study that evaluated the efficacy, safety, and pharmacodynamics of omaveloxolone in the treatment of patients with Friedreich ataxia.
In Part 2 of the study, patients were randomly assigned to receive omaveloxolone 150mg orally (n=51) or placebo (n=52). The final analysis included 40 omaveloxolone patients and 42 placebo patients. Findings showed that compared with placebo, omaveloxolone significantly improved neurological function, based on change in the modified Friedreich ataxia rating scale at 48 weeks (primary endpoint). The most common adverse events reported with omaveloxolone included headache, nausea, and fatigue.
“We are pleased with the FDA’s decision to grant Priority Review to our NDA for omaveloxolone for the treatment of patients with Friedreich ataxia in the United States,” said Warren Huff, Reata’s Chief Executive Officer. “We look forward to working with the FDA during the review process, and if approved, we are looking forward to a commercial launch in early 2023.”
The FDA is expected to make a decision on the application on November 30, 2022.
- Reata Pharmaceuticals announces FDA filing acceptance and Priority Review designation for the NDA for omaveloxolone for the treatment of patients with Friedreich’s ataxia. News release. May 26, 2022. https://www.businesswire.com/news/home/20220526005274/en/Reata-Pharmaceuticals-Announces-FDA-Filing-Acceptance-and-Priority-Review-Designation-for-the-NDA-for-Omaveloxolone-for-the-Treatment-of-Patients-with-Friedreich%E2%80%99s-Ataxia
- Lynch, DR, Chin, MP, Delatycki, MB, et al. Safety and efficacy of omaveloxolone in Friedreich ataxia (MOXIe study). Published online October 17, 2020. Annals of Neurology. doi.org/10.1002/ana.25934.