FDA Will Hold No Advisory Meeting on Omaveloxolone for FA, in Reversal
Agency's decision on Reata's oral treatment still expected early next year
In a reversal, the U.S. Food and Drug Administration (FDA) has informed Reata Pharmaceuticals that it will not hold an advisory committee meeting to discuss the company’s application for omaveloxolone, its oral treatment candidate for Friedreich’s ataxia.
The FDA’s reversal means that patients and the FA community will no longer have the opportunity to advocate before the advisory committee for omaveloxolone’s approval.
Meanwhile, the FDA’s decision on omaveloxolone is still expected in early 2023.
“Friedreich’s ataxia is a rare, genetic, debilitating, and degenerative neuromuscular disorder with no approved therapies, and we are committed to our goal of working to secure approval for omaveloxolone for patients living with this severe disease,” Warren Huff, Reata’s CEO, said in a press release announcing the FDA’s decision.
Reata submitted its application for omaveloxolone earlier this year. The FDA gave the application priority review and, at the time, informed Reata it was planning to hold an advisory committee meeting — a process in which the FDA convenes a group of experts in a field to review the available data for a potential therapy.
In a recent mid-cycle communication meeting, the FDA did not express any new concerns about safety, though the agency did raise concerns about the effectiveness of omaveloxolone, according to Reata. Following that meeting, Reata submitted new data supporting omaveloxolone to the FDA.
The agency then extended its review of the experimental treatment — a decision was originally expected by the end of November, but is now not anticipated until the end of February. Now, the FDA has announced it will not be holding an advisory committee before announcing a final decision early next year.
It’s not entirely clear what this current decision means for the future of omaveloxolone. This reversal could mean that, after reviewing the new data, the agency has already decided on whether or not to approve the therapy, and so sees no reason to convene a committee meeting — but there’s no way to know whether this could mean the therapy is definitely being approved, or definitely not, until the final decision is announced.
Omaveloxolone is designed to boost the activity of Nrf2, a protein that helps support the health of mitochondria that are dysfunctional in Friedreich’s ataxia.
Reata’s application is supported by data from a clinical trial called MOXIe (NCT02255435), which showed omaveloxolone outperformed a placebo on a standardized measure of disease progression after nearly a year of treatment. Long-term data out to nearly three years showed benefit for patients continuously on omaveloxolone compared with those initially given the placebo, and suggested a marked deviation from the untreated course of Friedriech’s ataxia.