Biogen Launches Phase 3 Study of SKYCLARYS for Pediatric FA
Biogen Initiates Groundbreaking Pediatric Study of SKYCLARYS
Biogen Inc. (NASDAQ: BIIB) has embarked on an innovative Phase 3 clinical trial known as the BRAVE study, which will explore the potential of omaveloxolone, marketed as SKYCLARYS, to treat Friedreich ataxia (FA) in children aged 2 to less than 16 years. This rare neurodegenerative disorder poses significant challenges for young patients, emphasizing the necessity for a focused therapeutic approach.
Pioneering Research for a Rare Disorder
The BRAVE study aims to address the critical and unmet medical needs of the pediatric FA population, a community that has historically faced a shortage of treatment options. By investigating the efficacy and safety of omaveloxolone, Biogen is demonstrating a commitment to developing solutions that could potentially alter the course of this debilitating disease for children in this age group.
Study Design and Insights
This multi-national Phase 3 trial will involve approximately 255 participants who exhibit symptoms of FA. The study will assess the drug's safety, efficacy, pharmacokinetics, and pharmacodynamics. Children will be randomly assigned to receive either omaveloxolone or a placebo daily for a duration of 52 weeks. After completing this stage, participants have the opportunity to join an open-label extension, receiving the active medication for an additional 52 weeks.
Recognizing the Urgent Need
Friedreich ataxia's early onset often correlates with faster disease progression, making the pediatric demographic particularly vulnerable. Dr. Stephanie Fradette, leading the Neuromuscular Development Unit at Biogen, highlighted this vital gap in care. "We are thrilled that the Phase 3 BRAVE study has begun, as our research targets a condition that has long been overlooked in younger patients. The feedback from the FA community has been invaluable in tailoring our study design," Dr. Fradette remarked.
Outcomes and Efficacy Measures
Part one of the BRAVE study focuses on the primary outcome measure, which is the change from baseline in the Upright Stability Score (USS). This score serves as an integral component of the modified Friedreich Ataxia Rating Scale (mFARS), making it a well-accepted measure of disease progress within the affected community.
Global Enrollment and Participation Opportunities
The BRAVE study has already commenced enrollment in the United States, with plans to expand to other countries, subject to regulatory approvals. The involvement of various experts ensures that the study design is comprehensive and rooted in the latest research findings. For families interested in participating, Biogen encourages discussions with their healthcare providers. Further inquiries can be made through Biogen’s Clinical Trials Center.
About SKYCLARYS
SKYCLARYS (omaveloxolone) is an oral medication designed for the treatment of Friedreich ataxia in older adolescents and adults, currently available in over 40 countries. The drug has received several designations from the U.S. FDA, including Orphan Drug designation, reflecting its potential to meet significant medical needs. The European Commission has similarly acknowledged it through the Orphan Drug designation specifically for its role in treating FA.
The Impact of Friedreich Ataxia
Friedreich ataxia is a rare genetic disorder that leads to progressive loss of coordination, muscle weakness, and fatigue, often manifesting during childhood. Due to the challenging nature of the disorder, most individuals diagnosed with FA may require the use of a wheelchair within a decade of symptom onset. Furthermore, the average lifespan of patients with this condition is significantly shortened, amplifying the critical need for effective treatment options.
About Biogen Inc.
Founded in 1978, Biogen has established itself as a leading biotechnology company focused on transforming lives through innovative medicine. With a robust pipeline of therapies and a commitment to addressing complex neurological disorders, the company continues to cultivate groundbreaking research that enhances patient outcomes and delivers significant shareholder value. Biogen’s proactive approach to understanding human biology further informs their commitment to developing treatments that resonate with patient needs.
Contact Information
The Biogen media relations team is available for inquiries and can be reached through Jack Cox at +1 781 464 3260 or through email. Investors can get in touch with Tim Power at +1 781 464 2442 for financial queries and corporate insights.
Frequently Asked Questions
What is the BRAVE study?
The BRAVE study is a global Phase 3 clinical trial initiated by Biogen to evaluate the efficacy and safety of omaveloxolone for treating Friedreich ataxia in children aged 2 to less than 16 years.
How many participants are involved in the BRAVE study?
Approximately 255 children diagnosed with Friedreich ataxia are expected to participate in the study.
What is omaveloxolone and its marketed name?
Omaveloxolone, marketed under the name SKYCLARYS, is an oral medication designed to treat Friedreich ataxia in older adolescents and adults.
What is the primary outcome measure for the study?
The primary outcome measure is the change from baseline in the Upright Stability Score (USS), which assesses balance and coordination in participants.
How can families participate in the BRAVE study?
Families interested in participating should consult their healthcare providers. Biogen also encourages direct inquiries through their Clinical Trials Center for more information.
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