On June 11th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, reported that KIZFIZO, temozolomide oral suspension, will be included in the MetroWILMS-1906 study (EudraCT: 2021-002540-67, NCT05384821) (Press release, ORPHELIA Pharma, JUN 11, 2024, View Source;utm_medium=rss&utm_campaign=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-nephrob [SID1234644263]). 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.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
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.