Biogen Inc. has announced the initiation of dosing in the BRAVE study, a pivotal Phase 3 clinical trial designed to evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) between the ages of 2 to 16 years. The global study represents a significant milestone in addressing the critical unmet medical need for pediatric patients with this rare neurodegenerative disorder.
Study Design and Objectives
The BRAVE study will evaluate omaveloxolone in approximately 255 children living with FA through a comprehensive two-part design. Part 1 consists of a 52-week, randomized, double-blind, placebo-controlled study where participants will be randomized 2:1 to receive omaveloxolone or placebo once daily. Both ambulatory and non-ambulatory participants may qualify for enrollment.
The primary outcome measure for Part 1 is the change from baseline in Upright Stability Score (USS), a subscale 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 lasting up to week 104, where all participants will receive omaveloxolone to further understanding of the drug's long-term effects.
Addressing Critical Unmet Need
"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," said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. "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."
Susan Perlman, M.D., Professor of Neurology and Director of the Ataxia Center at UCLA's David Geffen School of Medicine, emphasized the urgency of this research: "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."
Current Treatment Landscape
Omaveloxolone is currently marketed under the brand name SKYCLARYS in over 40 countries, including the United States and European Union, and represents the only approved product for FA in adults and adolescents aged 16 years and older. The drug has received multiple regulatory designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, as well as Orphan Drug designation from the European Commission.
Disease Burden and Prognosis
Friedreich ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder that represents the most common inherited ataxia. Early symptoms of FA, including progressive loss of coordination, muscle weakness and fatigue, typically appear in childhood and can overlap with other diseases. The disease progression is particularly severe, with most people living with FA requiring wheelchair use within 10-20 years of their first symptoms. 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.
Global Enrollment and Participation
The study design has been informed by previous studies and input from investigators, global medical experts and the FA community. Enrollment has initiated in the United States, with plans to open BRAVE study sites around the world pending final alignment with local regulators and ethics committees. The trial is registered as NCT06953583 on clinicaltrials.gov, and interested individuals can contact Biogen at clinicaltrials@biogen.com or call the Biogen Clinical Trials Center at 866-633-4636 for U.S. participants.
