Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia
Biogen's SKYCLARYS (omaveloxolone) receives European Commission approval for treating Friedreich’s ataxia, marking a significant advancement in rare disease treatment. The groundbreaking therapy demonstrated improved patient function in clinical trials, offering hope for patients with this debilitating condition.
The authorization of SKYCLARYS by the European Commission marks a pivotal advancement for the treatment of Friedreich’s ataxia (FA), a condition with previously limited therapeutic options. The approval is grounded on the MOXIe Part 2 trial, which demonstrated significant improvement in patients' mFARS scores, an assessment tool designed to evaluate the progression of ataxia and related symptoms. This is particularly noteworthy as FA is a rare disease and the development of treatments often faces challenges due to smaller patient populations and limited financial incentives.
From a research perspective, the approval also emphasizes the importance of post hoc analyses, such as the propensity-matched analysis mentioned, which can offer additional insights into a drug's long-term efficacy. However, it is crucial to recognize that such analyses are exploratory and should be interpreted with caution until validated by further studies.
The side effect profile of SKYCLARYS is also a critical aspect of its market acceptance. Common side effects like increased liver enzymes and decreased weight might affect patient compliance and quality of life, influencing the drug's real-world effectiveness. Ongoing pharmacovigilance will be essential to monitor these effects post-approval.
The EC's approval of SKYCLARYS represents not only a therapeutic milestone but also a significant economic event for Biogen and the rare disease market. The rarity of FA implies that SKYCLARYS will likely be classified as an orphan drug, which comes with specific market exclusivities and pricing implications. Orphan drugs typically command higher prices due to the lack of alternatives and the cost of research and development spread over a smaller patient base.
Furthermore, the establishment of early access programs in Germany and France indicates a strategic approach to market penetration, potentially allowing Biogen to recoup research and development costs more quickly. This strategy, while beneficial for early revenue generation, must be balanced against potential pressures from healthcare systems to justify the cost-effectiveness of new treatments, especially in the context of rare diseases with high treatment costs.
In the long term, the success of SKYCLARYS in the market will depend on its ability to demonstrate sustained clinical benefits and manage the overall cost of care for FA patients. This includes not only the direct costs of the medication but also potential reductions in associated healthcare expenses, such as hospitalizations or other supportive treatments.
The approval of SKYCLARYS in the European Union is a significant event that could influence Biogen's market position. It is the first approved treatment for FA in the EU, providing Biogen with a first-mover advantage in a new therapeutic area. The potential market size, while limited by the rarity of FA, is now essentially untapped and could provide a growth opportunity for the company.
Biogen's commitment to expanding access to SKYCLARYS through early access programs and engagement with regulatory authorities in other regions suggests a proactive approach to market expansion. The company's performance will be closely monitored by investors and industry analysts, as it could set a precedent for the commercial success of rare disease treatments in the global market.
It is also important to consider the role of patient advocacy groups, such as FARA and Euro-ataxia, in supporting drug development and approval processes. Their collaboration with Biogen could facilitate patient access and awareness, which are crucial for the uptake of new treatments. This dynamic between pharmaceutical companies and patient organizations can significantly impact market strategies and patient outreach efforts.
02/12/2024 - 04:06 PM
Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and neurodegenerative disorder Treatment with SKYCLARYS improved patient function compared to placebo Biogen is leveraging its expertise and capabilities in rare disease to bring this groundbreaking treatment to patients CAMBRIDGE, Mass., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.1
“In my clinical practice, I have seen the devastating impact that Friedreich’s ataxia has on patients and their families,” said Sylvia Boesch, M.D., MSc, Principal Investigator of the MOXIe study and Head of the Center for Rare Movement Disorders Innsbruck, Department of Neurology, Medical University Innsbruck, Austria. “Friedreich’s ataxia patients treated with SKYCLARYS in the clinical trial experienced important and clinically meaningful improvements for their daily lives. 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.”
Friedreich’s ataxia is the most common inherited ataxia.2,3 Early symptoms typically appear in childhood and include progressive loss of coordination, muscle weakness, and fatigue.4 As the disease progresses, people living with FA may also experience vision impairment, hearing loss, problems with speech and swallowing, diabetes, scoliosis, and serious heart conditions.5 Many people with FA use walking aids, and often require a wheelchair within 10-20 years following their diagnosis.2 Unfortunately, complications from FA contribute to a life expectancy of 37 years on average.5-7
“Biogen 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,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. “Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients. We sincerely thank the Friedreich’s ataxia community for their contributions that enabled the development of SKYCLARYS and made today’s approval possible.”
The EC approval of SKYCLARYS is based on efficacy and safety data from the placebo-controlled MOXIe Part 2 trial. At the end of the 48-week study, patients who received SKYCLARYS had significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores relative to placebo. All components of the mFARS assessment, including ability to swallow (bulbar), upper limb coordination, lower limb coordination, and upright stability, favored SKYCLARYS over placebo. Additional exploratory data was provided from a post hoc, propensity-matched analysis in which patients treated with SKYCLARYS in MOXIe (Extension) had lower mFARS scores at 3 years, as compared to a matched natural history group. The most common side effects are increased liver enzymes, decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.
“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,” said Jennifer Farmer, Chief Executive Officer of the Friedreich’s Ataxia Research Alliance (FARA). “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. We look forward to continuing our collaboration with the Friedreich’s ataxia community with the goal of expanding access where it is needed.”
“On behalf of Euro-ataxia and its members, I am delighted to welcome the European Commission approval of SKYCLARYS, a much-needed medicine for adults with Friedreich’s ataxia in EU countries,” said Andreas Nadke, President of Euro-ataxia. “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.”
For detailed product information, please see the Summary of Product Characteristics on the European Medicines Agency website at www.ema.europa.eu . Biogen is committed to working closely with all stakeholders to ensure that eligible European patients can have access to this treatment. Early access programs for SKYCLARYS are currently open in Germany and France, with plans to expand to additional countries where possible. SKYCLARYS is also approved for use in the United States, and Biogen is engaging with regulatory authorities in other regions.
About SKYCLARYS ® (omaveloxolone) ® (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older in the U.S. and European Union. SKYCLARYS received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration. The European Commission granted Orphan Drug designation in Europe to SKYCLARYS for the treatment of FA.
About Friedreich’s Ataxia 2,3 Early symptoms of FA, such as progressive loss of coordination, muscle weakness and fatigue, typically appear in childhood and can overlap with other diseases.4 Most people living with FA will need to use a wheelchair within 10-20 years of their first symptoms.2 The reported average age of death for FA patients is just 37 years old, although with appropriate and targeted care, individuals may live many years after confinement to a wheelchair.5-7
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References:
Friedreich’s Ataxia Research Alliance. “What is FA?” Available at: https://www.curefa.org/what-is-friedreichs-ataxia# . Accessed February 2024. National Institute of Neurological Disorders and Stroke. Friedreich Ataxia. Available at: https://www.ninds.nih.gov/health-information/disorders/friedreich-ataxia . Accessed February 2024. Schulz JB, Boesch S, Bürk K, Dürr A, Giunti P, Mariotti C, Pousset F, Schöls L, Vankan P, Pandolfo M. Diagnosis and treatment of Friedreich ataxia: a European perspective. Nat Rev Neurol. 2009 Apr;5(4):222-34. doi: 10.1038/nrneurol.2009.26. PMID: 19347027. Fogel BL, Perlman S. Clinical features and molecular genetics of autosomal recessive cerebellar ataxias. Lancet Neurol. 2007 Mar;6(3):245-57. doi: 10.1016/S1474-4422(07)70054-6. PMID: 17303531. Parkinson MH, Boesch S, Nachbauer W, Mariotti C, Giunti P. Clinical features of Friedreich’s ataxia: classical and atypical phenotypes. J Neurochem. 2013 Aug;126 Suppl 1:103-17. doi: 10.1111/jnc.12317. PMID: 23859346. Tsou AY, Paulsen EK, Lagedrost SJ, Perlman SL, Mathews KD, Wilmot GR, Ravina B, Koeppen AH, Lynch DR. Mortality in Friedreich ataxia. J Neurol Sci. 2011 Aug 15;307(1-2):46-9. doi: 10.1016/j.jns.2011.05.023. Epub 2011 Jun 8. PMID: 21652007. Corben LA, Collins V, Milne S, Farmer J, Musheno A, Lynch D, Subramony S, Pandolfo M, Schulz JB, Lin K, Delatycki MB; Clinical Management Guidelines Writing Group. Clinical management guidelines for Friedreich ataxia: best practice in rare diseases. Orphanet J Rare Dis. 2022 Nov 12;17(1):415. doi: 10.1186/s13023-022-02568-3. PMID: 36371255; PMCID: PMC9652828.
The approval marks a crucial milestone in rare disease treatment, providing the first approved therapy for Friedreich’s ataxia in the EU.
Common side effects include increased liver enzymes, decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.
Early symptoms include progressive loss of coordination, muscle weakness, fatigue, vision impairment, hearing loss, speech and swallowing problems, diabetes, scoliosis, and serious heart conditions.
The approval was based on efficacy and safety data from the placebo-controlled MOXIe Part 2 trial, showing significant improvements in modified Friedreich Ataxia Rating Scale (mFARS) scores.
Biogen aims to address the unmet medical need in Friedreich’s ataxia by providing the first treatment in Europe and engaging with stakeholders to ensure patient access.
