A liquid temozolomide for children with brain tumours wins EU backing at the second attempt
Orphelia Pharma's Kizfizo, an oral suspension of an old chemotherapy, has a positive CHMP opinion for malignant glioma from the age of one. Its first application, in neuroblastoma, was turned down last year.
In brief
The CHMP has backed Kizfizo, a temozolomide oral suspension, for children from age one with malignant glioma, after a 2025 rejection in neuroblastoma.
- The CHMP adopted a positive opinion on Kizfizo, a 40 mg/ml temozolomide oral suspension, on 15 October 2026.
- The paediatric indication starts at one year of age; the reference product, Temodal, is authorised for children from three.
- Orphelia's earlier application, for relapsed or refractory high-risk neuroblastoma, received a negative opinion in February 2025.
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Kizfizo, a ready-to-use oral suspension of temozolomide developed by the French company Orphelia Pharma. The opinion was adopted on 15 October 2026 and published the following day, alongside the committee's monthly highlights.
Temozolomide is hardly new. Temodal, the reference product, has been authorised in the EU since January 1999, as hard capsules and as a powder for infusion. What Kizfizo adds is a 40 mg/ml liquid, and with it a paediatric indication that reaches younger children than the existing product does.
What the opinion covers
The recommended indications mirror Temodal's: adults with newly diagnosed glioblastoma multiforme, given alongside radiotherapy and then as monotherapy, and patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, that recurs or progresses after standard therapy. The difference lies in the age threshold. Temodal's recurrent glioma indication starts at three years; Kizfizo's covers children from the age of one.
Kizfizo was reviewed as a hybrid application, a route that relies partly on the reference product's clinical data. The EMA said studies showed satisfactory quality and bioequivalence to Temodal. The medicine held an orphan designation for glioma during development, and the agency will now review whether it can be maintained.
Why a liquid matters
Capsules are a poor fit for small children, many of whom cannot swallow them, and a fixed range of capsule strengths makes fine dose adjustment harder in the smallest patients. Temodal's only other form is a powder for infusion. A licensed oral liquid, measured by syringe, gives prescribers an authorised option for children who would otherwise depend on unlicensed workarounds.
The value of the product lies less in the molecule than in the age group it can now reach.
The first attempt
The route to approval was not direct. Orphelia first sought authorisation for Kizfizo in children with relapsed or refractory high-risk neuroblastoma, drawing on pharmacokinetic data from the TEMOkids study and efficacy and safety evidence from the BEACON-Chemo and Retro-TMZ studies. On 27 February 2025 the CHMP issued a negative opinion, finding the data did not sufficiently show clinical benefit. Laurent Martin, Orphelia's chief pharmaceutical affairs officer, said at the time that the company was "deeply disappointed" and would explore other pathways.
The glioma application took a different approach, anchoring the product to Temodal's established indications rather than seeking a new one. That made the evidential burden one of formulation and bioequivalence rather than fresh efficacy.
What comes next
The opinion now passes to the European Commission for a decision. For sponsors, the episode illustrates a recurring pattern in paediatric oncology: age-appropriate formulations of old cytotoxics can reach the market more readily by extending established uses than by opening new indications in rare childhood cancers, where controlled data are hardest to generate.
