KIMOZO (product in development)
Temozolomide oral suspension, 40mg/ml
Product in development for the treatment of relapsed or refractory neuroblastoma.
KIMOZO 40 mg/ml is a ready-to-use and taste-masked oral suspension of temozolomide to be used for the treatment of pediatric patients with relapsed or refractory neuroblastoma.
Temozolomide may also be useful in treatment protocols for other rare solid pediatric cancers.
KIMOZO is presented in a kit containing 1 vial filled with 20 ml of temozolomide oral suspension (40mg/ml) and 1 dosing oral syringe graduated up to 5 ml with a 0.1 ml dosing increment.
KIMOZO is not approved yet.
ORPHELIA Pharma is developing KIMOZO as monotherapy or in combination with a specific DNA inhibitor topoisomerase I (irinotecan or topotecan) for the treatment of patients aged 12 months and above with:
- high-risk refractory neuroblastoma or presenting an insufficient response to induction chemotherapy,
- recurrent high-risk neuroblastoma after at least partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.
The EMA has recognized that capsules are not adapted to the pediatric population and are not accepted in very young children(1). Temozolomide hard-gel capsules are not appropriate for the pediatric population, especially for children under 6 years of age who cannot swallow them. Parents and caregivers may be required to open the capsules and mix the content with soft food for administration.
In the Draft Inventory of paediatric therapeutic needs for paediatric oncology published in 2014, the EMA underlined the need of an “age-appropriate solid or liquid formulation” of temozolomide. As a ready-to-use and taste-masked oral suspension of temozolomide, KIMOZO fully responds to this need(2).
KIMOZO has nearly completed its clinical development program, which includes:
- The “Bioequivalence Study Between Temozolomide Oral Suspension (Kimozo®) and Temodal® Capsules” is an open label, randomized, crossover, multi center, 2-period study in 30 male/female adult patients with primary CNS malignancies. The study demonstrated bioequivalence between Kimozo® and Temodal® (NCT04467346, completed).
- TEMOkids is a pharmacokinetic, acceptability and safety study in pediatric patients aged 1 to less than 18 years, in need of temozolomide. It is an international open-label, non-randomized, prospective, single-arm phase 1 study to determine the pharmacokinetic (PK), the acceptability and the safety of KIMOZO in the pediatric population. You can visit the platform clinicaltrials.gov for more details (NTC04610736).
- RetroTMZ is a retrospective study in 6 centers in 3 countries (France, Spain, Switzerland) with 196 children with refractory or relapsed high-risk neuroblastoma diagnosed between 2004 and 2017, followed-up to 02/02/2021, receiving single-agent or combined TMZ, assessing efficacy and tolerability of TMZ (MR004 N° 2207313).
KIMOZO received an Autorisation d’Accès Précoce (AAP) for the treatment of patients aged 1 to 6 years of age and in patients older than 6 years of age, who are unable to swallow temozolomide in capsule form suffering with relapsed or refractory neuroblastoma in March 2022.
KIMOZO may also be available upon named patient approvals from French Authorities (ANSM) for other rare solid pediatric cancers under the Autorisation d’Accès Compassionnel (AAC) scheme, in patients aged 1 to 6 years of age and in patients older than 6 years of age, who are unable to swallow temozolomide in capsule form.
Please visit ansm.sante.fr for more information.
- Ducray et al., A bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules inpatients with primary central nervous system malignancies, ASCO 2022 congress: Abstract and Poster
- Lemarchand et al., Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures, SIOP 2022 congress: Abstract and Poster
- Annereau et al., Development of a Hospital Compounded, Taste-Masked, Temozolomide Oral Suspension and 5-Year Real-Life Experience in Treating Paediatric Patients, Pharmaceuticals, 2022.
- Metayer et al., SIOP 2019 Abstract (Temozolomide as a single agent or in combination for patients with high-risk refractory or relapsed neuroblastoma: excellent tolerance and sustained disease responses)