Plans Progress for Approval of Omaveloxolone

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Reata Pharmaceuticals is planning to ask the U.S. Food and Drug Administration (FDA) to approve omaveloxolone to treat people with Friedreich’s ataxia (FA) early in 2022, following the completion of a preliminary meeting with the agency.

“Omaveloxolone could be the first drug approved for the treatment of Friedreich’s ataxia — actually the first drug approved for any ataxia,” Susan Perlman, MD, professor of neurology at the University of California, Los Angeles, said in a press release.

“While not a cure, if approved, Friedreich’s ataxia would finally become a treatable disease, something the Friedreich’s ataxia community has been working towards for a long time,” Perlman added.

Reata and the FDA recently held a preliminary meeting to discuss the company’s planned new drug application (NDA) — the formal request of approval — for omaveloxolone. Reata is planning to seek standard approval of omaveloxolone for FA, and the company is not planning additional clinical studies prior to submission.

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At the meeting, the FDA indicated the appropriate approval pathway would be a matter of review after the NDA is submitted. Also, given the seriousness of FA and the lack of treatment for the disease, the FDA indicated that, pending reviews, Reata may be allowed to submit additional clinical and nonclinical data even after the NDA is officially submitted.

“We are pleased with the outcome of our recent pre-NDA meeting and that we have a path to submit our NDA in the first quarter of 2022,” said Warren Huff, president and CEO of Reata.

Omaveloxolone (RTA-408) is an investigational oral therapy designed to activate a protein called Nrf2. This protein is a transcription factor, helping to regulate how genes are “read” in cells and increasing the activity of certain genes. In particular, Nrf2 boosts the activity of genes that are important for the function of mitochondria — the energy-generating cellular organelles that are impaired in FA — and in combatting oxidative stress, a type of cellular damage thought to contribute to the progression of the disease.

Results from a two-part placebo-controlled Phase 2 clinical trial called MOXIe (NCT02255435) showed that treatment with omaveloxolone significantly improved multiple measures of neurological function, and was generally safe and well-tolerated.

“The MOXIe Part 2 study with omaveloxolone is the first to demonstrate a significant improvement in neurological function in patients with Friedreich’s ataxia,” Perlman said.

Based on the findings, tens of thousands of people have signed a petition from the Friedreich’s Ataxia Research Alliance (FARA) calling on the FDA to approve omaveloxolone for the treatment of FA.

“We remain committed to our goal of working with the FDA to secure regulatory approval for omaveloxolone as quickly as possible for patients with this devastating disease,” Huff concluded.