Biogen 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.

“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.