Otsuka Pharmaceutical Europe and Lundbeck have announced that the European Commission has approved Rxulti (brexpiprazole) for the treatment of schizophrenia in adolescents aged 13 years and older.

The approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency in January 2025.

The decision was based on a six-week, randomised, double-blind placebo-controlled and active-referenced trial involving 316 adolescent patients. The study evaluated the efficacy and safety profile of brexpiprazole, showing greater reductions in symptom severity, as measured by the Positive and Negative Syndrome Scale (PANSS) total score compared with placebo.

Brexpiprazole, dosed at 2-4 mg/day, was generally well tolerated with a safety profile consistent with that observed in adult patients with schizophrenia.

Andy Hodge, CEO at Otsuka Pharmaceutical Europe, said: “The prognosis for adolescence-onset schizophrenia is poor compared with adult-onset schizophrenia and can be associated with more chronic and severe symptoms. 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.”

Johan Luthman, EVP and Head of Research & Development at Lundbeck, said: “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 is an atypical oral antipsychotic taken once a day, modulating serotonin and dopamine systems. It was previously approved in the European Union in 2018 for the treatment of adult schizophrenia.

Orphelia Pharma is exploring alternative regulatory pathways for KIZFIZO after receiving a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on 27 February 2025. The company is determined to secure access to this treatment for paediatric cancer patients.

The CHMP’s decision came despite the extensive data submitted by Orphelia Pharma, which did not sufficiently demonstrate KIZFIZO’s clinical benefit. Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphelia Pharma, expressed deep disappointment, acknowledging the impact on the neuroblastoma community.

“We are deeply disappointed by the CHMP’s opinion, especially after the extensive efforts and substantial data we submitted to support the clinical benefit of KIZFIZO,”  said Martin. “Considering the important unmet medical need, we are currently exploring all available options to make KIZFIZO available to the paediatric patients.”

Orphelia Pharma will continue to make KIZFIZO available to pediatric patients under compassionate use or early access programmes and ongoing clinical trials, subject to agreement by relevant authorities.

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid paediatric formulation for children with relapsed or refractory high-risk neuroblastoma. It was granted Early Access Authorisation by the French authorities in March 2022 and has received Orphan Drug Designation from the EMA and the FDA.

The pharmacokinetics of KIZFIZO in children have been evaluated in the TEMOkids study. Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma includes the BEACON-Chemo and Retro-TMZ studies.