Products and pipeline


West Syndrome

Vigabatrin scored soluble tablets, 100mg & 500mg

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. 

Unmet medical need in pediatric patients

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) 






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.

Kimozo® (product in development)

Relapsed or refractory neuroblastoma

Temozolomide oral suspension, 40mg/ml

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.