Orphelia Pharma applies to EMA its drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma

Orphelia Pharma, a pharmaceutical company focused on pediatric and orphan medicines, has filed a centralized Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for KIZFIZO, the first oral liquid formulation of temozolomide.

KIZFIZO (temozolomide oral suspension, 40 mg/ml), known as Ped-TMZ or KIMOZO during its clinical development and ongoing early access programs, is designed specifically for use in the treatment of children with relapsed or refractory neuroblastoma, oncology indications with a very poor prognosis. This oral suspension, which is taste-masked, was developed for children: it allows a precise dose to be administered orally or via a nasogastric tube in a small volume. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last six years.

Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma, said: “KIZFIZO fills an unmet medical need for a drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma. This formulation aims to avoid the use of non-age-appropriate dosage forms mixed with a drink or food, which may expose caregivers to a cytotoxic molecule without full control of the dose actually delivered to the patient.”

Prof. Fabrice Barlesi, CEO of Gustave Roussy, said: “The application for a drug marketing authorization of KIZFIZO is excellent news for the children living with cancer and their families. It underscores Gustave Roussy’s commitment to playing a leading role in the development of medicines for children and to curing all of them in the future.”