FDA Extends Review of Omaveloxolone in Friedreich’s Ataxia

The updated PDUFA date is Feb. 28, 2023, and the planned advisory committee meeting is on hold pending review of NDA amendments.

The FDA has extended by three months the review of Reata Pharmaceuticals’ omaveloxolone for the treatment of patients with Friedreich’s ataxia, a rare, genetic, degenerative neuromuscular disorder.

The company has submitted updated data from the MOXIe Extension study, including delayed-start analysis using a March 2022 data cut-off and a new propensity-matched analysis using patient data from the Clinical Outcome Measures in Friedreich’s Ataxia Study as controls. Additionally, Reata submitted an analysis of the relevance of Nrf2, the target of omaveloxolone, to the pathophysiology of Friedreich’s ataxia.

These submissions were provided as confirmatory evidence of the results of the MOXIe Part 2 study in response to concerns raised by the FDA during the mid-cycle communication meeting. The FDA determined that these submissions were a major amendment to our NDA and extended the Prescription Drug User Fee Act date to provide time for a full review of the new data and analyses.

The updated PDUFA date is Feb. 28, 2023, and the planned advisory committee meeting is on hold pending review of NDA amendments.

“We are pleased with the FDA’s decision to review the new information we recently provided to the Division,” Warren Huff, Reata’s chief executive officer, said in a press release. “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 severe disease that has no approved therapies.”

Patients with Friedreich’s ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their 20s. It impacts about 5,000 children and adults in the United States and 22,000 individuals globally. Currently, there are no approved therapies for the treatment of Friedreich’s ataxia.

Omaveloxolone is an oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

The FDA has granted orphan drug, fast track, and rare pediatric disease designations to omaveloxolone for the treatment of Friedreich’s ataxia.