Orphelia to explore alternative regulatory pathways to secure access for pediatric cancer patients following confirmed CHMP negative opinion
Paris and Lyon, France, March 12th, 2025 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed on 27 February 2025 a negative opinion regarding the Marketing Authorization Application for KIZFIZO (temozolomide oral suspension) for the treatment of patients with relapsed or refractory high-risk neuroblastoma, further to the request for re-examination of the opinion submitted by the company in December 2024. The CHMP considered that the clinical benefit of KIZFIZO was not sufficiently demonstrated, preventing the establishment of a positive benefit-risk balance.
“We are deeply disappointed by the CHMP’s opinion, especially after the extensive efforts and substantial data we submitted to support the clinical benefit of KIZFIZO,” said Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphelia Pharma. “We understand that this outcome is also a disappointment for the neuroblastoma community and the families of children suffering from this cancer. Considering the important unmet medical need, we are currently exploring all available options to make KIZFIZO available to the pediatric patients.”
In the meantime, Orphelia Pharma will, subject to agreement by the relevant authorities, continue to make KIZFIZO available to pediatric patients under compassionate use or early access programs as well as to patients participating into ongoing clinical trials.
About KIZFIZO® 40 mg/ml
KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed/designed specifically for use in the treatment of children with relapsed or refractory high-risk neuroblastoma, which carries 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 neuroblastoma as monotherapy or in combination with irinotecan or topotecan.
KIZFIZO has received Orphan Drug Designation (ODD) from the EMA and the FDA, the formulation is covered by granted patents and pending applications in Europe and the US.
The pharmacokinetics of KIZFIZO in children have been evaluated in TEMOkids, a European multicenter population pharmacokinetic acceptability and safety study in pediatric patients in need of temozolomide (NCT04610736).
Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma submitted in the application includes in particular:
- BEACON-Chemo, a sub-analysis of the chemotherapy arms of the BEACON study, a prospective randomized phase II study in refractory or relapsed neuroblastoma. This study was sponsored by Birmingham University (UK).
- Retro-TMZ, a multicenter descriptive, retrospective study, assessing the efficacy and tolerability of temozolomide in children with refractory or relapsed neuroblastoma. This study was conducted by Gustave Roussy (France).
About Neuroblastoma
Neuroblastoma is the most common extracranial cancer in early childhood, with approximately 900 new cases diagnosed per year in the European Union. It almost exclusively affects children under five, with a median age at diagnosis of 18 months. Neuroblastoma has a wide diversity of clinical outcomes, which is reflected in the risk stratification. Approximately 40% of patients have the high-risk disease and often face a poor response to first line induction therapy or later relapse. There remains a high unmet need for relapsed or refractory neuroblastoma patients and the best therapeutic strategy is still an intensive area of research. Temozolomide is the standard chemotherapy and is therefore an essential part of the treatment armamentarium for these patients.
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.