Skyclarys becomes first treatment for FA to win approval in Europe
As with US approval, oral therapy indicated for EU patients 16 and older
The European Commission has approved Skyclarys (omaveloxolone) for people with Friedreich’s ataxia (FA) ages 16 and older, making the treatment the first to win approval for FA in the European Union.
The decision comes just a few months after a committee of the European Medicines Agency (EMA) issued a positive recommendation for Skyclarys. It also follows less than a year after the therapy became the first to win approval for FA in the U.S.
“The European Commission approval of Skyclarys is a significant milestone toward expanding global access, bringing the first approved treatment to the Friedreich’s ataxia community in the EU,” Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance (FARA), said in a press release from Biogen, which markets the therapy.
“FARA is grateful to all the researchers, clinical sites, individuals with Friedreich’s ataxia and their families, patient organizations, Biogen and the European Medicines Agency for the research, drug development and advocacy efforts that led to this approval,” Farmer said.
Biogen, which acquired Skyclarys from its original developer last year, now plans to work with the EU member states to get patients access to the newly approved medication, the company said.
EU approval of Skyclarys based on positive findings of MOXie trial
Priya Singhal, MD, Biogen’s head of development, said the company is “proud to add Skyclarys to our portfolio of medicines and address a significant unmet need by bringing the first treatment to people living with Friedreich’s ataxia in Europe.”
“Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients,” Singhal added. “We sincerely thank the Friedreich’s ataxia community for their contributions that enabled the development of Skyclarys and made today’s approval possible.”
Friedreich’s ataxia, an inherited, progressive disease, is characterized by problems with cellular energy-making structures called mitochondria. These problems lead to reduced energy production in cells as well as inflammation and a type of cell damage called oxidative stress.
Skyclarys is designed to boost energy production, limit inflammation, and lessen oxidative stress by targeting a protein called nuclear erythroid 2-related factor 2 (NrF2). That proteing helps regulate the activity of genes in cells.
With this approval, there is optimism within the community that Skyclarys has the potential to usher in a new era in the management of Friedreich’s ataxia.
The EU approval was based on data from a Phase 2 study called MOXie (NCT02255435) that enrolled more than 170 people with FA. The trial included three parts: an initial dose-finding step, a placebo-controlled study, and an open-label extension.
Data from the placebo-controlled part of the study, which compared Skyclarys with a placebo for about one year, showed that patients given the once-daily medication had better neurological function after a year. Those results indicated that Skyclarys was able to slow the progression of FA in patients.
Findings from the extension study, out to three years, have consistently suggested that neurological disease progression is slower for FA patients on Skyclarys than would be expected in untreated individuals
The therapy’s most common side effects include elevated liver enzymes, loss of appetite, weight loss, nausea, vomiting, and diarrhea. Headache, fatigue, throat and back pain, muscle spasms, and influenza also were commonly experienced as side effects to the medication.
“Friedreich’s ataxia patients treated with Skyclarys in the clinical trial experienced important and clinically meaningful improvements for their daily lives,” said Sylvia Boesch, MD, principal investigator of the MOXIe study at Medical University Innsbruck, in Austria.
“With this approval, there is optimism within the community that Skyclarys has the potential to usher in a new era in the management of Friedreich’s ataxia,” Boesch said.
Andreas Nadke, president of the nonprofit Euro-ataxia, an umbrella organization with 15 member groups, called Skyclarys “a much-needed medicine for adults with Friedreich’s ataxia in EU countries.”
“Our member’s patient groups have been working and waiting for this day for many years, and we firmly believe that this will be a successful and gratifying beginning in the treatment of Friedreich’s ataxia,” Nadke said.
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