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ORPHELIA Pharma announces the publication of two scientific articles on KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma

9 January 2024

Paris and Lyon, January 9th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announces the publication of two scientific articles about KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma, which is under registration review under centralised procedure by the EMA.

The first article, entitled Dispensing Oral Temozolomide in Children: “Precision and Stability of a Novel and Ready to Use Liquid Formulation in Comparison with Capsule Derived Mixtures”, published in Pharmaceutics, demonstrates that the clinical practice of administering temozolomide capsules (Temodal) by mixing the content with food or drinks results in significant underexposure compared to administering the ready-to-use KIZFIZO 40 mg/ml oral suspension.

Tests for the accuracy of the administered dose and stability of temozolomide were performed with the capsule contents mixed with food vehicles (apple juice, applesauce, cream, milk and mashed potatoes) and compared to an equivalent dose of KIZFIZO. The dose delivered was systematically lower with capsule-based preparations, regardless of the vehicle used.

In addition, certain food vehicles (dessert cream, milk, mashed potatoes) had a significant effect on the chemical degradation of temozolomide, with the significant increase of its hydrolysis product, amino-imidazole-carboxamide (AIC).

The second article, also published in Pharmaceutics, as : A Multicenter Randomized Bioequivalence Study of a Novel Ready-to-Use Temozolomide Oral Suspension vs. Temozolomide Capsules”, demonstrates the bioequivalence between KIZFIZO oral suspension and the capsule form of temozolomide (Temodal).

In conclusion, the administration of KIZFIZO, which is bioequivalent with the capsule form of temozolomide, avoids the misuse associated with the administration of capsules in the paediatric population. This ready to use oral liquid formulation, which is taste masked and ensures the chemical stability of temozolomide, accurately delivers the prescribed dose.

About KIZFIZO® 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. 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.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received an Orphan Drug Designation from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

About Orphelia Pharma

Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world.

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