EMA Backs Oral Suspension for Malignant Brain Tumors

At its October 2026 meeting, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has recommended Kizfizo (temozolomide, Orphelia Pharma) 40 mg/mL oral suspension for adults with newly diagnosed glioblastoma and for adults and children aged 1 year or older with recurrent or progressive malignant glioma. Kizfizo is a hybrid medicine of Temodal, the capsule formulation authorized in the EU since January 26, 1999. It contains the same active substance. The new product differs in being a 40 mg/mL suspension, and it extends approval to patients aged 1 year to less than 3 years.New Option for Malignant Brain Tumor Kizfizo is indicated for adults with newly diagnosed glioblastoma, administered alongside radiotherapy and subsequently as monotherapy. It is also indicated for children aged 1 year or older, adolescents, and adults with recurrent or progressive malignant glioma, including glioblastoma and anaplastic astrocytoma, following standard therapy.Kizfizo contains temozolomide as an active substance, which is an antineoplastic alkylating agent that damages tumor cell DNA, disrupting DNA repair and ultimately causing cell death. Evidence Supports Use The CHMP recommendation is supported by bioequivalence data and clinical evidence confirming efficacy in both adult and pediatric populations. A pivotal phase 3 trial enrolled 573 patients with newly diagnosed, histologically confirmed glioblastoma. Participants received radiotherapy alone or radiotherapy plus daily temozolomide, followed by six cycles of adjuvant temozolomide. At a median follow-up of 28 months, median overall survival was 14.6 months with combined radiotherapy and temozolomide, compared with 12.1 months with radiotherapy alone. The hazard ratio for death was 0.63 (95% CI, 0.52-0.75; P < .001), and the 2-year survival rate was 26.5% vs 10.4%, respectively. Bioequivalence was assessed in a randomized, crossover study of 36 adults with glioma, of whom 30 completed the trial. The oral suspension and capsule formulations met standard bioequivalence criteria. In adult and pediatric patients with recurrent or progressive malignant glioma, temozolomide improved progression-free survival compared with procarbazine.Safety Profile and Prescribing Considerations The most frequent adverse events observed with Kizfizo align with the known safety profile of temozolomide, including nausea, vomiting, constipation, diarrhea, anorexia, headache, fatigue, convulsions, hemiparesis, aphasia or dysphasia, alopecia, and rash. In the pivotal phase 3 trial, grade 3 or 4 hematologic toxicity occurred in 7% of patients receiving combined treatment. Treatment should be prescribed by physicians experienced in managing brain tumors.Regulatory Next Steps Kizfizo received orphan medicinal product designation for glioma in the European Union on August 22, 2025. The designation provides regulatory support for developing medicines intended to treat rare conditions but does not itself authorize marketing or patient access.Following the CHMP recommendation, the European Commission will consider whether to grant marketing authorization. The EMA will also review the available information to determine whether Kizfizo’s orphan designation can be maintained. If authorization is granted, detailed prescribing recommendations will be published in the product information in all official EU languages.