Orphelia Pharma is exploring alternative regulatory pathways for Kizfizo (temozolomide) following a negative opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
The latest decision follows another negative opinion for Orphelia’s marketing authorisation application (MAA) in November 2024. The CHMP determined that Kizfizo failed to demonstrate sufficient clinical benefit to establish a positive benefit-risk balance. The agency cited modest response rates and the lack of convincing evidence that Kizfizo offered significant advantages over existing therapies for relapsed/refractory neuroblastoma.
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In response to the initial negative opinion, Orphelia requested a re-examination of the decision in December 2024. However, after a re-evaluation on 27 February 2025, the CHMP upheld its initial stance, stating that the committee’s concerns were not resolved. The company shared this update in a press release today (12 March).
Orphelia emphasised that it will continue to make Kizfizo available to paediatric patients under compassionate use or early access programmes as well as to patients participating in ongoing clinical trials.
“Considering the important unmet medical need, we are currently exploring all available options to make Kizfizo available to the paediatric patients,” said Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma in the 12 March announcement.
Neuroblastoma is a type of cancer that forms in certain types of nerve tissue, mostly affecting children under the age of five. Neuroblastoma is the second most common solid tumour in children after brain tumours, according to the UK-based charity The Children & Young People’s Cancer Association (CCLG). Kizfizo was designed as a hybrid medicine similar to MSD’s chemotherapy agent Temodal but is formulated as a liquid for easier administration, as many babies and children cannot take oral tablets.
Orphelia presented clinical data from studies enrolling 102 patients up to 21 years old. The Phase I TEMOkids (NCT04610736) and Ped-TMZ studies (NCT04467346) demonstrated safety, pharmacokinetics, and efficacy. The ongoing Phase I/II BEACON2 study is evaluating Kizfizo in combination with chemotherapies irinotecan or topotecan.
Originally indicated exclusively for the treatment of glioblastoma in adults in the US, temozolomide has been used off label to treat neuroblastoma patients for years. The drug has been approved for treating glioblastoma in adults and children in Europe since 2010. Kizfizo was granted early access authorisation by the French authorities in March 2022 and has received orphan drug designation from the EMA and the US Food and Drug Administration (FDA).
The only approved drug in Orphelia’s portfolio is Kigabeq (vigabatrin), its paediatric formulation of vigabatrin, which has an EU authorisation as a monotherapy for West syndrome and for use as a combination treatment for resistant partial epilepsy.
