Otsuka Pharmaceutical Europe and Lundbeck announced today that the European Commission (EC) has approved Rxulti (brexpiprazole) for the treatment of schizophrenia in adolescents aged 13 years and older. This approval extends the drug's previous indication, which was limited to adult patients since its initial European approval in 2018.
The EC decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued in January 2025.
Clinical Evidence Supporting Approval
The approval was based on compelling data from a rigorous 6-week, randomized, double-blind, placebo-controlled trial involving 316 adolescent patients. The study demonstrated that brexpiprazole at doses of 2-4 mg/day significantly reduced symptom severity compared to placebo, as measured by the Positive and Negative Syndrome Scale (PANSS) total score.
Importantly, the safety profile observed in adolescents was consistent with that previously established in adult patients with schizophrenia. The medication was generally well-tolerated across the adolescent population studied.
Addressing a Critical Treatment Gap
Schizophrenia affects approximately 24 million people worldwide, or about 1 in 300 individuals. The condition frequently emerges during late adolescence and early adulthood, with onset typically occurring earlier in males than females.
"The prognosis for adolescence-onset schizophrenia is poor compared with adult-onset schizophrenia and can be associated with more chronic and severe symptoms," noted Andy Hodge, CEO at Otsuka Pharmaceutical Europe. "We welcome the EC decision to extend the indication for brexpiprazole to include adolescents aged 13 years and older, providing young people in Europe with another much-needed treatment option."
Schizophrenia in adolescents presents unique challenges, as younger patients may be particularly vulnerable to medication side effects. The condition is characterized by delusions, hallucinations, and disordered cognition, often causing significant distress and impairment in personal, educational, and social functioning.
Mechanism of Action
Brexpiprazole is an atypical oral antipsychotic administered once daily. Its pharmacological profile involves modulatory activity at both serotonin and dopamine systems, combining partial agonist activity at serotonergic 5-HT1A and dopaminergic D2 receptors with antagonist activity at serotonergic 5-HT2A receptors. The drug also demonstrates antagonist activity at noradrenergic α1B/2C receptors.
This unique mechanism is believed to contribute to the medication's efficacy in managing schizophrenia symptoms while maintaining a favorable tolerability profile.
Industry Perspectives
Johan Luthman, EVP and Head of Research & Development at Lundbeck, emphasized the significance of this regulatory milestone: "Today marks a major milestone for young patients, caregivers, and families navigating the complexities of schizophrenia. This approval is testament to our commitment and unwavering support to lessen the disease burden for patients and caregivers in the EU by providing a treatment option with proven efficacy and tolerability."
Brexpiprazole was discovered by Otsuka and is being co-developed and co-commercialized under a collaboration and license agreement between Otsuka Pharmaceutical Europe Ltd. and H. Lundbeck A/S.
Public Health Impact
Schizophrenia ranks among the top 15 leading causes of disability worldwide. For adolescents with this condition, effective and well-tolerated treatment options are crucial for managing symptoms and improving long-term outcomes.
The expanded indication for brexpiprazole represents an important addition to the therapeutic armamentarium available to clinicians treating adolescent schizophrenia in Europe, potentially helping to address the significant unmet needs in this vulnerable patient population.
