Biogen Initiates Phase 3 Pediatric Study of Omaveloxolone for the Treatment of Friedreich Ataxia

Rhea-AI Summary

Biogen (BIIB) has initiated the BRAVE study, a global Phase 3 clinical trial evaluating omaveloxolone in children aged 2 to <16 years with Friedreich ataxia (FA). The study will enroll approximately 255 participants, randomized 2:1 to receive omaveloxolone or placebo for 52 weeks. The primary outcome measure is the Upright Stability Score (USS), a subscale of the modified FA rating scale. Omaveloxolone is currently marketed as SKYCLARYS® in over 40 countries for FA treatment in patients 16 and older. The study consists of two parts: a 52-week placebo-controlled phase followed by an open-label extension up to week 104. This trial addresses a critical unmet need, as early-onset FA patients often experience more aggressive disease progression, and no approved treatments currently exist for the pediatric population.

Positive

• Expansion of potential treatment to pediatric population (ages 2 to <16) where there are currently no approved treatments
• Large-scale study with approximately 255 participants
• SKYCLARYS is already approved and marketed in over 40 countries for patients 16 and older
• Study design informed by previous studies and input from investigators, global medical experts and FA community

Negative

• None.

Insights

Biotech/Pharmaceutical Clinical Researcher

Biogen's Phase 3 pediatric trial for SKYCLARYS represents significant expansion opportunity for an already-approved rare disease treatment.

Biogen has initiated the BRAVE Phase 3 study evaluating omaveloxolone (marketed as SKYCLARYS®) in children with Friedreich ataxia (FA) aged 2 to <16 years. This represents a critical expansion of their clinical program, as SKYCLARYS is already approved in over 40 countries for adults and adolescents aged 16+ with FA.

The trial design shows careful consideration of pediatric endpoints. The primary outcome measure will be the Upright Stability Score (USS), a subscale specifically recognized as the most sensitive measure of disease progression in children with FA. The study's 2:1 randomization ratio (drug:placebo) and planned open-label extension demonstrate a thoughtful approach to both gathering efficacy data and ensuring all participants eventually receive treatment.

From a strategic perspective, this pediatric development makes strong clinical sense. FA typically begins in childhood, with earlier symptom onset associated with faster progression. By targeting children specifically, Biogen is addressing the most vulnerable patient population with the highest unmet need. The large sample size (approximately 255 children) suggests the company's significant commitment to this program.

The study's inclusion of both ambulatory and non-ambulatory participants acknowledges the disease's spectrum and increases the potential patient population if approved. This pediatric program, if successful, would expand SKYCLARYS from a treatment for teens/adults to a comprehensive solution across most of the FA age spectrum, significantly strengthening Biogen's rare disease portfolio.

• Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with Friedreich ataxia, a rare neurodegenerative disorder
• BRAVE study will explore the potential of omaveloxolone to address the critical unmet need of the pediatric FA population
• Omaveloxolone is currently marketed under the brand name SKYCLARYS^® for the treatment of adults and adolescents aged 16 years and older affected by Friedreich ataxia
  

CAMBRIDGE, Mass., June 18, 2025 (GLOBE NEWSWIRE) --  Biogen Inc. (Nasdaq: BIIB) announced the initiation of dosing in the BRAVE study, a global Phase 3 clinical trial. The BRAVE study will evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) between the ages of 2 to <16. Both non-ambulatory and ambulatory participants may qualify for the study. Participants will be randomized 2:1 to receive omaveloxolone or placebo once a day for 52 weeks before having the opportunity to move into the open-label extension (OLE). Currently, omaveloxolone is commercialized under the brand name SKYCLARYS^® in over 40 countries, including in the U.S. and the European Union, and is the only approved product for FA in adults and adolescents aged 16 years and older.

“Recognizing the symptoms of Friedreich ataxia typically begin in childhood, and earlier onset of symptoms is associated with faster disease progression, there is tremendous unmet need in the pediatric community. Building on the work of Reata we have been urgently advancing the pediatric development plan for omaveloxolone and are thrilled that the Phase 3 BRAVE study has now begun,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. “We are immensely grateful for the input from the entire FA community that has helped shape the design of this important study.”

The BRAVE study will evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of omaveloxolone in approximately 255 children living with FA. Part 1 is a 52 week, randomized, double-blind, placebo-controlled study, designed to evaluate efficacy of omaveloxolone compared to placebo. The primary outcome measure for Part 1 is change from baseline in Upright Stability Score (USS), a subscale that is recognized by the FA community as the most sensitive means of measuring disease progression in children living with FA. USS is part of the validated modified FA rating scale (mFARS). Part 2 will be an open-label extension (up to week 104) where all participants will receive omaveloxolone to further our understanding of the long-term effects of the drug.

“Early onset patients often have the most aggressive and fast progressive form of Friedreich ataxia and through the BRAVE study we aim to determine the potential safety and efficacy of omaveloxolone for children living with the disease. This vulnerable population faces significant unmet need, with no approved treatments currently available,” said Susan Perlman, M.D., Professor of Neurology and Director of the Ataxia Center, David Geffen School of Medicine at UCLA.

The design of this Phase 3 study has been informed by previous studies and input from investigators, global medical experts and the FA community. Enrollment has initiated in the United States and we plan to open BRAVE study sites around the world pending final alignment with local regulators and ethics committees. Individuals interested in participating in this study should speak with their healthcare provider. They can also email Biogen at clinicaltrials@biogen.com. Individuals located in the U.S. can also call the Biogen Clinical Trials Center at 866-633-4636. Both clinicaltrials.gov (NCT06953583) and Biogen Trial Link (Biogen Trial Link) will be updated as more information about the BRAVE study becomes available.

About SKYCLARYS^® (omaveloxolone)
SKYCLARYS® (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 over 40 countries, including 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.

Please click here for Important Safety Information and full Prescribing Information for SKYCLARYS^® (omaveloxolone) in the U.S. or visit your respective country’s product website.

About Friedreich Ataxia
Friedreich ataxia (FA) is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder. It is the most common inherited ataxia.^1,2,3 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

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube.

Biogen Safe Harbor 
This news release contains forward-looking statements including relating to the potential benefits, safety and efficacy of SKYCLARYS; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of Friedreich’s ataxia; the potential of Biogen’s commercial business and pipeline programs, including Friedreich’s ataxia; and risks and uncertainties associated with drug development and commercialization. These statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. You should not place undue reliance on these statements.

Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. 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References:

1. Friedreich’s Ataxia Research Alliance. “What is FA?” Available at: https://www.curefa.org/what-is-friedreichs-ataxia#. Accessed February 2024.
2. National Institute of Neurological Disorders and Stroke. Friedreich Ataxia. Available at: https://www.ninds.nih.gov/health-information/disorders/friedreich-ataxia. Accessed February 2024.
3. 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.
4. 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.
5. 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.
6. 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.
7. 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.
   
   

MEDIA CONTACT: Biogen Jack Cox + 1 781 464 3260 public.affairs@biogen.com

INVESTOR CONTACT: Biogen Tim Power +1 781 464 2442 IR@biogen.com

FAQ

What is the purpose of Biogen's BRAVE Phase 3 study for SKYCLARYS (omaveloxolone)?

The BRAVE study aims to evaluate the efficacy and safety of omaveloxolone in children aged 2 to <16 years with Friedreich ataxia, addressing an unmet need in pediatric patients.

How many participants will be enrolled in BIIB's BRAVE Phase 3 trial?

The BRAVE study will enroll approximately 255 children living with Friedreich ataxia.

What is the primary outcome measure for the BRAVE study?

The primary outcome measure is the change from baseline in Upright Stability Score (USS), a subscale of the modified FA rating scale (mFARS) recognized as the most sensitive means of measuring disease progression in children with FA.

How long will Biogen's BRAVE Phase 3 trial last?

The study consists of a 52-week placebo-controlled phase (Part 1) followed by an open-label extension up to week 104 (Part 2).

In which countries is SKYCLARYS currently approved for Friedreich ataxia?

SKYCLARYS is currently approved and marketed in over 40 countries, including the United States and European Union, for patients aged 16 years and older.