Biogen Obtains European Commission Approval for SKYCLARYS® (omaveloxolone), the Initial Therapy for Friedreich’s Ataxia
Tuesday, February 13, 2024
Biogen Inc. (Nasdaq: BIIB) has announced that SKYCLARYS® (omaveloxolone) has received authorization from the European Commission (EC) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. This approval represents a significant milestone as it marks the first sanctioned treatment for this rare, genetic, progressive neurodegenerative disease within the European Union.
Dr. Sylvia Boesch, M.D., MSc, Principal Investigator of the MOXIe study and Head of the Center for Rare Movement Disorders Innsbruck at the Department of Neurology, Medical University Innsbruck, Austria, shared insights into the challenges faced by patients and their families dealing with Friedreich’s ataxia. She highlighted notable improvements observed in patients treated with SKYCLARYS during clinical trials, instilling hope within the community about the potential of SKYCLARYS to revolutionize the management of Friedreich’s ataxia.
Friedreich’s ataxia, known as the most common inherited ataxia, typically presents symptoms such as progressive loss of coordination, muscle weakness, and fatigue, often manifesting in childhood. As the disease progresses, individuals may also experience vision and hearing impairments, speech and swallowing difficulties, diabetes, scoliosis, and severe heart conditions. Many FA patients rely on walking aids, with a wheelchair becoming necessary within 10-20 years of diagnosis. Unfortunately, FA-related complications lead to an average life expectancy of 37 years.
Biogen, emphasized the company's dedication to addressing the unmet needs of Friedreich’s ataxia patients in Europe with the approval of SKYCLARYS. The approval is based on compelling efficacy and safety data from the placebo-controlled MOXIe Part 2 trial, demonstrating significant improvements in modified Friedreich Ataxia Rating Scale (mFARS) scores among patients treated with SKYCLARYS compared to placebo.
Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance (FARA), expressed gratitude for the collaborative efforts that led to the EC approval of SKYCLARYS, emphasizing the importance of expanding access to treatment within the FA community.
Andreas Nadke, President of Euro-ataxia, welcomed the EC approval of SKYCLARYS on behalf of Euro-ataxia and its members, acknowledging the years of anticipation within patient groups and expressing confidence in SKYCLARYS as a significant step forward in FA treatment.
Biogen is dedicated to ensuring eligible European patients have access to SKYCLARYS, with early access programs already underway in Germany and France and plans for further expansion. SKYCLARYS is also approved for use in the United States, with regulatory engagement ongoing in other regions.
SKYCLARYS® (omaveloxolone) is an oral, once-daily medication indicated for the treatment of FA in adults and adolescents aged 16 years and older in both the U.S. and the European Union. It has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. FDA, as well as Orphan Drug designation from the European Commission.
Friedreich’s ataxia (FA) is a rare, genetic, life-shortening neuromuscular disorder characterized by progressive degeneration. Early symptoms typically emerge in childhood, leading to significant disability and reduced life expectancy.
Source: globenewswire.com
