Reata Accepts FDA’s Suggestion of Pre-NDA Meeting for Omaveloxolone

The U.S. Food and Drug Administration (FDA) has asked Reata Pharmaceuticals to request a preliminary meeting to discuss submitting a new drug application (NDA) for omaveloxolone, an investigational therapy for Friedreich’s ataxia (FA).

Reata was due to hold a Type C meeting (unrelated to NDA requests) with the FDA’s Division of Neurology Products 1, but the division determined that, after reviewing the company’s Type C meeting briefing material, a pre-NDA meeting was more appropriate.

“We welcome the opportunity to have a pre-NDA meeting regarding our omaveloxolone development program for the treatment of patients with FA,” Warren Huff, Reata’s president and CEO, said in a press release.

Reata now plans to withdraw its Type C meeting request and submit one for a pre-NDA meeting as soon as practicable.

The announcement follows a recent petition from the Friedreich’s Ataxia Research Alliance (FARA) and signed by more than 74,000 people worldwide, asking that Reata submit an NDA for omaveloxolone (omav) on an urgent basis and that the FDA consider its approval based on existing evidence.

The existing evidence in this case, came from the MOXIe Phase 2 clinical trial (NCT02255435). Although the trial met its main goal of showing improved neurological function, the FDA declared the results were not strong enough to merit regulatory approval.

A separate analysis involving participants who received a placebo in the first and second parts of MOXIe, before transitioning to omav in the trial’s open-label extension study, also failed to convince the FDA.

Given the lack of therapies available for FA and the progressive nature of the disorder, the FDA decision prompted the FA community to question exactly how robust trial findings need to be to win FDA approval. To that end, the petition included a request for the FDA to clarify sufficient evidence exactly.

The FDA decision represented a significant setback for the FA community, both because of how difficult it is to recruit participants for rare disease trials and because of the outsize effects that even small therapeutic benefits have in patients’ daily lives.

Shandra Trantham, a participant in the MOXIe trial who is currently part of the long-term extension study, described the effects of these small benefits in an interview with Friedreich’s Ataxia News in February.

She reported having more energy throughout the day and walking better since taking omav. She also described watching the difference in disease progression grow between her and other friends with FA, who are not participating in the trial.

“I know that every time I see them, they progress a lot,” she said, “and … if I look at myself, I’m really not progressing.”

Omaveloxolone restores a measure of function to the mitochondria — the cell’s energy generators that are impaired in FA — by activating Nrf2, a protein that controls the activity of several genes involved in mitochondrial function and energy production.

“We look forward to working with the FDA on our goal of securing the regulatory review and approval necessary to make omaveloxolone available to patients with FA,” said Huff.