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ORPHELIA Pharma to collaborate with Oscar Lambret clinical center to study KIZFIZO®, the first pediatric drinkable formulation of temozolomide, for the treatment of refractory or relapsed nephroblastoma (Wilms tumor)

11 June 2024

Paris and Lyon, June 11th, 2024 Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, is pleased to announce that KIZFIZO, temozolomide oral suspension, will be included in the MetroWILMS-1906 study (EudraCT: 2021-002540-67, NCT05384821). MetroWILMS is a French, multicentric, non-randomized, Phase 1-2 clinical trial to study the efficacy and safety of metronomic chemotherapy, including temozolomide, for the treatment of refractory or relapsed nephroblastoma (Wilms tumor). MetroWILMS is sponsored by the Oscar Lambret clinical center on behalf of the Société Française des Cancers de l’Enfant (SFCE), Nephroblastoma committee.

Pediatric renal (kidney) tumors account for 4%-11% of all childhood cancers, the most common of which being nephroblastoma or Wilms tumor. It affects young children, most of the cases being diagnosed before 5 years of age. Despite a good prognosis, 10 to 15 % of patients present a refractory Wilms tumor or experience relapse and bear a dismal prognosis.

As part of the MetroWILMS protocol, KIZFIZO (under the name KIMOZO currently in use in France) will be administered for the treatment of refractory/relapsed Wilms tumors (after 1 or 2 lines of treatment or after 1 line for high risk relapse for which there would not be any curative therapy), using a metronomic schedule and in combination with other drugs (vincristine, irinotecan, etoposide and cis retinoic acid). MetroWILMS will include 28 pediatric patients, with a recruitment planned until October 2028.

“There is a major clinical need for an oral liquid form of temozolomide in various pediatric oncology indications, including in refractory or relapsed Wilms tumor”, said Hélène Sudour-Bonnange, onco-pediatrician at Oscar-Lambret clinical center and principal investigator of MetroWILMS trial. “We believe that KIZFIZO oral suspension is especially well suited to treat our young patients.”

“Refractory or relapsed nephroblastoma is a very rare indication, but this is well in line with Orphelia’s missions to help pediatric patients with rare cancers”, said Jérémy Bastid, chief medical officer at Orphelia Pharma. “Should MetroWilms demonstrate a clinical benefit, we could consider expanding the label of KIFZIZO in this indication”.

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 (under the trade name KIMOZO) 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 Oscar Lambret

Oscar Lambret Centre, founded in 1955, is part of the UNICANCER group, which brings together the 18 cancer treatment centres, private health establishments of collective interest (ESPIC), exclusively dedicated to care, research and teaching in cancerology and ensuring patients access to all treatments and care, regardless of financial resources and without any additional cost. The Centre was certified by the French National Authority for Health (HAS) in 2023.

The Centre’s ambition is to accelerate innovation in cancer care for all patients in the North of France, whether in terms of treatment, the development of scientific research projects or the training of tomorrow’s specialists.

Oscar Lambret center is the comprehensive cancer center of North of France. This hospital is labelled for management of pediatric cancers and labelled for running early phase clinical trials. The “Direction de la Recherche Clinique et de l’Innovation” (DRCI) of Centre Oscar Lambret is certified ISO-9001:2015 since 2018. Oscar Lambret center is currently the sponsor of 20 clinical trials.

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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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