Products and pipeline


West Syndrome

Vigabatrin scored soluble tablets, 100mg & 500mg

Kigabeq® is a pediatric form of vigabatrin developed in breakable soluble tablets with two strengths (500mg and 100mg) for the treatment of infantile spasms (West syndrome). West syndrome is an extremely serious disease in infancy which progresses into major encephalopathy if untreated and for which vigabatrin is a key component of the treatment armamentarium.

The drug was granted an European Marketing Authorization in September 2018, 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. Kigabeq® fully responds to this need.

Kigabeq® allows precise and convenient dosage of vigabatrin

Bioequivalent to Sabril™ 500 mg granules for oral solution,
100 mg and 500 mg scored soluble tablets with fast water dispersion (< 1 min),
50 mg dosing steps for dose-to-body weight adjustment and titration of the drug,
Possibility to obtain concentrated solutions,
Easy handling for care givers,
No wastage of the medicine.


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, 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 drinkable suspension, 40mg/ml

Kimozo® is a new pharmaceutical form of temozolomide (oral drinkable suspension).

Kimozo® is a pediatric formulation currently under 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 dismal prognosis.

Kimozo® is the first oral pediatric formulation of temozolomide

Kimozo is a ready-to-use oral suspension administered using an oral syringe. A dose up to 200 mg of temozolomide can be administered with one syringe.

  • Age-appropriate formulation
  • Small volumes
  • Accurate dose
  • Taste-masked
  • Avoids caregiver exposure to a cytotoxic drug
  • Avoids inappropriate handling of capsules for young children