Marketing Authorization Application for KIZFIZO® accepted for review by the EMA for the treatment of malignant glioma
London, UK, March 30, 2026 – Orphalan, a pharmaceutical company dedicated to the development of innovative treatments for rare disease, today announces that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for KIZFIZO® (temozolomide oral suspension, 40 mg/ml). The formal procedure for the review commenced on 22 January 2026.
KIZFIZO® is the first oral liquid formulation of temozolomide specifically designed for paediatric patients and patients with dysphagia (swallowing difficulties). This formulation allows for precise dosing and is intended to provide an adapted treatment option for both paediatric and adult populations where solid dosage forms are not appropriate.
The claimed indication for KIZFIZO® submitted in the application is for the treatment of:
- Children from the age of one year to less than seven years, and dysphagic children above seven years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy.
- Adult dysphagic patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy (RT) and subsequently as monotherapy treatment.
“The acceptance of this application by the EMA is a significant milestone for Orphalan and, more importantly, for the patients we serve,” said Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphalan. KIZFIZO® is designed to meet a clear unmet medical need. By developing a 40 mg/ml oral suspension, we aim to make available a new medicinal product designed for paediatric or dysphagic patients, ensuring they can receive their medication accurately and safely.”
About KIZFIZO® 40 mg/ml KIZFIZO® (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid formulation of temozolomide. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma, now part of Orphalan.
About Malignant Glioma and Glioblastoma Multiforme
Malignant gliomas are fast-growing, aggressive tumours of the brain and spinal cord. Glioblastoma multiforme (GBM) is the most common and most aggressive type of malignant glioma in adults. These tumours are difficult to treat because they grow rapidly and invade nearby brain tissue. Standard therapy typically involves surgery followed by radiation and chemotherapy. For young children, the oral suspension is the only appropriate oral dosage form of temozolomide for treating patients and for older patients, dysphagia (difficulty swallowing) is a symptom occurring in approximately one third of the patients, with especially high prevalence as the disease progresses, making the administration of solid oral medications a significant challenge.1
About Orphalan
Founded in 2011, Orphalan is an international pharmaceutical company dedicated to the development and commercialisation of orphan drugs. Born of a successful collaboration with AGEPS and Hôpital Lariboisière, Orphalan has built a global presence with authorised treatments currently available in 38 countries. Orphalan’s ambition is to make innovative orphan medicines accessible to as many patients as possible through a patient-centred approach and close collaboration with healthcare professionals.
Following the acquisition of Orphelia Pharma on 1 December 2025, a company focused on the development of medicines for rare and serious paediatric diseases, Orphalan has significantly expanded its therapeutic portfolio and expertise. KIZFIZO® was a key product brought into the group through this acquisition. This integration combines Orphalan’s global reach with specialized research in neurology and oncology, furthered by key academic partnerships including collaborations with Gustave Roussy.
[1] IJzerman-Korevaar, M., Snijders, T.J., de Graeff, A., Teunissen, S.C.C.M. and de Vos, F.Y.F. (2018) ‘Prevalence of symptoms in glioma patients throughout the disease trajectory: a systematic review’, Journal of Neuro-Oncology, 140(3), pp. 485–496. doi: 10.1007/s11060-018-03015-9.
