Following Biogen’s $7.3 billion acquisition of Reata Pharmaceuticals in July 2023, the drug on Monday was approved in the European Union for treating the rare genetic disorder that causes progressive damage to the nervous system.
Pictured: Biogen’s office in Cambridge, Massachusetts/iStock, hapabapa
The European Commission on Monday approved Biogen’s Skyclarys (omaveloxolone) for the treatment of the rare genetic disorder Friedrich’s ataxia in patients aged 16 years and above.
Skyclarys is the first treatment approved for Friedrich’s ataxia (FA) in the European Union, according to Biogen’s announcement. In March 2023, Skyclarys got U.S. approval, becoming the first-ever FDA-approved therapy for FA.
Monday’s approval in Europe will allow Biogen to “address a significant unmet need” for patients, Priya Singhal, head of development at Biogen, said in a statement. “Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access to patients.”
The European Commission’s approval is based on data from the MOXIe Part 2 trial, a randomized, double-blinded and placebo-controlled study assessing Skyclarys in patients with genetically confirmed FA. The trial administered Skyclarys once daily at a 150-mg dose and assessed its efficacy primarily through the modified Friedreich’s Ataxia Rating Scale (mFARS), which evaluates patients’ neurological function.
MOXIe results showed that Skyclarys was significantly superior to placebo in terms of all mFARS components, including the ability to swallow, upper and lower limb coordination and upright stability.
Biogen also provided additional exploratory data from the MOXIe (Extension) study, which showed that three years after Skyclarys treatment mFARS scores remained better than natural history controls.
FA is an ultra-rare, progressive neuromuscular disease caused by a mutation in the FXN gene, which encodes for the protein frataxin, normally found in the mitochondria and a key player in the cell’s energy production.
The disease typically arises during childhood and initially manifests as fatigue, muscle weakness and progressive loss of coordination. Patients with FA eventually depend on wheelchairs within 10 to 20 years of their first symptoms and die at an average age of 37 years old.
Skyclarys is an oral small molecule drug that targets the underlying pathway of FA by activating the Nrf2 transcription factor, which restores mitochondrial function and helps mitigate oxidative stress. The drug was first developed by Reata Pharmaceuticals, which Biogen acquired in July 2023 for $7.3 billion in a bid to boost its neurological and rare disease portfolios.
In addition to Skyclarys, the Reata acquisition gave Biogen the muscular atrophy treatment Spinraza (nusinersen) and the amyotrophic lateral sclerosis therapy Qalsody (tofersen).
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
