Reata scores first FDA approval in its 21-year history, pushing rare disease drug over finish line

On Rare Disease Day, Reata celebrated in appropriate fashion with its first drug approval in the company’s 21-year history.

The FDA has signed off on Reata’s Skyclarys (omoveloxolone) for the treatment of Friedreich’s ataxia (FA), a hereditary neurological disorder that strikes during adolescence and leaves patients wheelchair-bound and often cuts short their lives.

Skyclarys becomes the first drug approved for the condition, which affects roughly 5,000 who have been diagnosed in the United States and 22,000 worldwide. Reata expects to make the medicine available by the end of the second quarter of this year.

The drug will carry a list price of $370,000 annually, the company said Tuesday in a conference call. Jefferies analyst Maury Raycroft has pegged U.S. sales potential at $400 million by 2030.

Reata has submitted an application for approval in Europe, with a plan to launch there in early 2024.

With news of the approval, Reata’s stock shot up from $31.10 per share at close on Tuesday to $84.03 by mid-morning on Wednesday.

The Plano, Texas-based biotech earned the FDA endorsement with evidence from just one trial—which is unusual but bodes well for the future of approvals for diseases that affect relatively few people.

In 2020, the FDA turned thumbs down on the drug based on results from the Moxie phase 3 study of 103 patients, which showed treatment led to significant improvement in walking at Week 48 over placebo. But regulators needed to see more and asked the company to conduct another trial.

Over the last three years however—following up with patients from the same study—Reata has shown regulators what they needed to see in terms of safety and neurological function measures such as speaking, swallowing and standing.

Skyclarys is an oral, once-daily Nrf2 transcription factor that binds to the Keap1 gene, restoring mitochondrial function and reducing inflammation. It is not the first small molecule that Reata has brought before regulators.

Last year, to the day, the FDA rejected the company’s bardoxolone for the treatment of Alport syndrome, which leads to chronic kidney disease. That decision came after an advisory committee voted 13-0, recommending that it not be blessed.

Skyclarys appeared destined to a similar fate in August of last year when the FDA delayed the timeline for its approval by three months to further hash out the risk/benefit profile of the drug.

“It is an important milestone for Reata because it is the culmination of 15 years of scientific research and clinical development on our Nrf2 platform,” Reata CEO Warren Huff said during the conference call.

The approval covers patients 16 and older. The company said that it is opening an ongoing study in Europe to younger patients, with hopes of getting a label expansion to those age 6 and older.