Products and pipeline
Kigabeq®
West Syndrome
Kigabeq® is a pediatric form of vigabatrin developed as breakable soluble tablets with two strengths (500mg and 100mg) for the treatment of West syndrome (a subtype of infantile spasms) as monotherapy and for the treatment of resistant partial epilepsy (focal onset seizures) in combination with other antiepileptic medicinal products.
West syndrome is a rare disorder characterized by the association of clusters of axial spasms, psychomotor retardation and an hypsarrhythmic interictal EEG pattern. It is the most frequent type of epilectic encephalopathy. It may occur in otherwise healthy infants and in those with abnormal cognitive development.1
Vigabatrine is considered as first-line therapy for patients with West syndrome.
Kigabeq® was granted a European Marketing Authorization in September 2018 and ORPHELIA Pharma is the Marketing Authorization Holder.
The EMA, in the “Revised priority list for studies into off-patent pediatric medicinal products” published in 2014 underlined the need of an “age-appropriate formulation” of vigabatrin. With its 2 strengths and the scored tablets, Kigabeq® fully responds to this need.
1. Reference: Infantile spasms syndrome (Nov.2021)

Ivozall®
Relapsed or refractory acute lymphoblastic leukaemia
Clofarabine concentrate for solution for infusion, 1 mg/ml
Ivozall® is a concentrate of clofarabine for the treatment of relapsed or refractory acute lymphoblastic leukemia. The drug was granted an European Marketing Authorization in November 2019 and ORPHELIA Pharma is the Marketing Authorization Holder.
Acute lymphoblastic leukaemia is a type of cancer that affects white blood cells. It progresses quickly and aggressively and requires immediate treatment. Although it is rare, acute lymphoblastic leukaemia is the most common type of leukaemia that affects children. About 85% of the cases that affect children happen in those younger than 15 (mostly between the ages of 0 and 5).
Ivozall® : medicinal product containing clofarabine authorised under the European centralized procedure
Ivozall® is a concentrate for solution for infusion. Each mL of concentrate contains 1 mg of clofarabine and the product is supplied in 20 ml-vials. Clofarabine is an essential medicine for the treatment of relapsed or refractory Acute Lymphoblastic Leukaemia (ALL) in children. Ivozall® is the first generic form of clofarabine to be authorised under the European centralized procedure.

ORPHELIA Pharma exploite le médicament :
IVOZALL 1mg/ml, solution à diluer pour perfusion (clofarabine)
Pour toute déclaration liée à un effet indésirable de nos médicaments, veuillez contacter le service Pharmacovigilance à l’adresse suivante :
pv@orphelia-pharma.eu
Pour toutes questions sur nos médicaments, veuillez contacter le service Information Médicale à l’adresse suivante :
infomed@orphelia-pharma.eu
Pour toute réclamation qualité, veuillez contacter le service Qualité à l’adresse suivante : pqc@orphelia-pharma.eu
Contact téléphonique : 01.42.77.08.18
Consulter la base de données publique des médicaments : Medicament.gouv.fr / IVOZALL 1 mg/ml, solution à diluer pour perfusion
Kimozo® (product in development)
Relapsed or refractory neuroblastoma
Kimozo® is a new pharmaceutical form of temozolomide (oral suspension).
Kimozo® is a pediatric formulation currently in clinical development. It is a ready-to-use and taste-masked suspension for oral administration. ORPHELIA Pharma is currently developing Kimozo® for the treatment of relapsed or refractory neuroblastoma, a rare pediatric cancer of poor prognosis.

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 patients with primary CNS malignancies. The study demonstrated bioequivalence between Kimozo® and Temodal®. You can visit the platform clinicaltrials.gov for more details.
TEMOkids study 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 aged 1 year and over.
You can visit the TEMOkids study website: www.temokids.eu and the platform clinicaltrials.gov for more details.
Kimozo® est distribué par ORPHELIA Pharma en France en accès précoce ou accès compassionnel depuis le 1er juin 2022.
Pour toute déclaration liée à un effet indésirable de nos médicaments, veuillez contacter le service Pharmacovigilance à l’adresse suivante :
pv@orphelia-pharma.eu
Pour toutes questions sur nos médicaments, veuillez contacter le service Information Médicale à l’adresse suivante :
infomed@orphelia-pharma.eu
Pour toute réclamation qualité, veuillez contacter le service Qualité à l’adresse suivante : pqc@orphelia-pharma.eu