Biogen Launches BRAVE Phase 3 Trial Testing SKYCLARYS® In Children With Friedreich Ataxia

Biogen has officially begun dosing participants in its global Phase 3 BRAVE clinical trial, which will assess the safety and efficacy of omaveloxolone in children aged 2 to under 16 with Friedreich ataxia (FA). The trial is open to both ambulatory and non-ambulatory patients. Participants will be randomized in a 2:1 ratio to receive either omaveloxolone or placebo once daily for 52 weeks, after which they will have the opportunity to enter an open-label extension (OLE). Omaveloxolone, marketed as SKYCLARYS®, is currently approved for use in patients aged 16 and above and is the only authorized treatment for FA in the U.S. and Europe.

“Recognizing the symptoms of Friedreich ataxia typically begins 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. We are immensely grateful for the input from the entire FA community that has helped shape the design of this important study,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen.

The BRAVE study aims to enroll approximately 255 pediatric patients and is divided into two parts. The first part is a 52-week double-blind, placebo-controlled phase to evaluate the treatment’s efficacy based on changes in the Upright Stability Score (USS), a subscale of the validated modified Friedreich Ataxia Rating Scale (mFARS) recognized as a sensitive measure of disease progression in children. The second part, an open-label extension, will run up to week 104 and allow all participants to receive omaveloxolone to better understand its long-term impact.

“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 study design reflects insights gained from earlier trials as well as collaboration with global clinicians and the FA patient community. Biogen has initiated enrollment in the United States and plans to expand to additional international sites pending regulatory and ethics board approvals. The trial marks a significant step forward in addressing unmet needs for younger FA patients.