Orphelia Pharma explores other pathways for paediatric cancer treatment

Company seeks new direction after negative CHMP opinion on KIZFIZO

Orphelia Pharma is exploring alternative regulatory pathways for KIZFIZO after receiving a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on 27 February 2025. The company is determined to secure access to this treatment for paediatric cancer patients.

The CHMP’s decision came despite the extensive data submitted by Orphelia Pharma, which did not sufficiently demonstrate KIZFIZO’s clinical benefit. Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphelia Pharma, expressed deep disappointment, acknowledging the impact on the neuroblastoma community.

“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 Martin. “Considering the important unmet medical need, we are currently exploring all available options to make KIZFIZO available to the paediatric patients.”

Orphelia Pharma will continue to make KIZFIZO available to pediatric patients under compassionate use or early access programs and ongoing clinical trials, subject to agreement by relevant authorities.

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation for children with relapsed or refractory high-risk neuroblastoma. It was granted Early Access Authorization by the French authorities in March 2022 and has received Orphan Drug Designation from the EMA and the FDA.

The pharmacokinetics of KIZFIZO in children have been evaluated in the TEMOkids study. Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma includes the BEACON-Chemo and Retro-TMZ studies.