Skyclarys now available to patients with Friedreich’s ataxia

Reata Pharmaceuticals announced that Skyclarys is now commercially available for patients older than 16 years who have Friedreich’s ataxia, a rare degenerative neuromuscular disorder that manifests at a young age.

The FDA clearance of the Prior Approval Supplement to update the drug substance specification paved the way for its availability, the company said.

“Skyclarys’ commercial availability is an enormous milestone for Friedreich’s ataxia (FA) patients and their families who were instrumental in making this first and only treatment for FA a reality,” according to Reata chief commercial officer and executive vice president Dawn C. Bir, in a statement provided to Healio. “This devastating, rare and progressive neuromuscular disease impacts approximately 5,000 people in the United States.”

Bir said in the first 2 months of the Feb. 28 FDA approval of once-daily Skyclarys (omaveloxolone), the company’s patient access services center received more than 500 patient start forms from more than 250 neurologists, primary care physicians and other health care providers. She noted that Reata has worked for 15 years to validate its Nrf2 activator platform, and now the company is focused on accessibility to the treatment.

FA manifests at a young age, passed down from both parents, Reata told Healio in a previous interview during the American Academy of Neurology meeting.

“The diagnosis often occurs in the teens, and then approximately 10 to 15 years later they’re in a wheelchair,” she said. “The approval is huge for a patient community that has waited so long.”

MOXIe, a placebo-controlled, multicenter, randomized trial involving 103 patients with FA, showed that those treated with omaveloxolone over 48 weeks experienced less physical impairment, Bir said. Patients took three 50 mg pills once daily before eating.

Bir said Reata activated patient communication and began sales and promotional efforts a week after the February approval.

“The patient community is thrilled to finally have a treatment option,” she said. “They feel like they now have something they can do; they’re not just watching their disease progress. This is a highly engaged, younger patient population. These patients have been seeing neurologists, but without an approved treatment, a primary care doctor is quarterbacking their care. We believe with an approved treatment, many will return to their neurologist for [care].”

Reata considers patient access a core strategy, Bir said. “We believe every FA patient is deserving of the only treatment. We can provide guidance to payers on how they approve policies. We’ve ensured out-of-pocket costs will be minimal or nothing. If that’s not available, they can have patient assistance or free drug.”

She said Reata is working to make Skyclarys available to younger patients as soon as possible.

“It’s diagnosed when kids are young, and they start to display symptoms like clumsiness,” Bir said. “It could take 3 years until a diagnosis is made. It’s usually diagnosed in the early teens.”

She concluded: “Reata was founded over 20 years ago with a focus on unmet need and high science. It’s been a long journey. To see this FDA approval has been a milestone. It represents the culmination of over 15 years of research and development and provides validation of our Nrf2 activator platform.

“It’s an honor to bring a drug to the market that has the potential to create a change for the betterment of patients in an area with such significant unmet need.”

Editor’s note: On July 8, 2023, this story was updated to clarify the MOXIe study results.