ORPHELIA Pharma’s mission is to improve the treatment of life-threatening pediatric diseases and orphan conditions. Our aim is to provide meaningful advances to children and patients suffering from rare diseases through the development of efficient, reliable and easy-to-use medicines. Our projects focus on the development of existing or new drugs for the treatment of new pediatric indications or orphan diseases as well as novel pediatric formulations of approved drugs.
Our current pipeline covers pediatric neurology (epilepsy), rare pediatric oncology and onco-hematology conditions.
The first pediatric formulation of vigabatrin
Kigabeq® : a pediatric formulation of the antiepileptic drug vigabatrin
The evaluation of Kigabeq® by the European Medicine Agency (EMA) has been completed and the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Kigabeq® on 26 July 2018, recommending the issuance of a European marketing autorisation. Kigabeq® has been exclusively developed for young children (1 – 7 years) and will thus benefit from a Pediatric Use Marketing Autorisation (PUMA).
Kigabeq® is a pediatric formulation of the antiepileptic drug vigabatrin which inhibits the GABA transaminase enzyme. Vigabatrin has been marketed throughout Europe since 1989 and in the USA since 2009 (Sabril®). Vigabatrin is currently approved for the treatment of infantile spasms and pharmaco-resistant focal-onset seizures (partial seizures).
Infantile spasms is a rare and devastating form of epileptic encephalopathy with peak incidence at about 6 months of age. It is an orphan disease with about 2,500 new annual cases in Europe. If left untreated, the prognosis is poor with deleterious effect on brain maturation and cognitive development.
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. The formulation of Kigabeq® fully responds to this need.
Kigabeq® allows precise and convenient dosage of vigabatrin
- Bioequivalent to Sabril™ sachet,
- 100 mg and 500 mg scored soluble tablets with fast water dispersion (< 30 seconds),
- 50 mg dosing steps for dose-to-bodyweight adjustment and titration of the drug,
- Possibility to obtain concentrated solutions,
- Easy handling for care givers,
- No wastage of the medicine.
Kigabeq® was granted a Paediatric Use Marketing Authorization (PUMA) from the European Commission on September 20st, 2018.
Products in development
Preclinical and clinical stage programs
ORP-010 / Ivozall®
ORP-010 is a drug used to treat the acute lymphoblastic leukemia. The development has been completed and Ivozall® was granted an European Marketing Authorization in November 2019.
ORP-005 / Kimozo®
Kimozo® is a paediatric formulation of temozolomide, presented as a taste-masked suspension for oral administration. ORPHELIA Pharma is currently developing Kimozo® for the treatment of acute or relapsed neuroblastoma, a rare paediatric cancer of dismal prognosis. Kimozo® is currently under clinical development.
ORP-011 is a new formulation of a medicine used in acute leukemia, both in children and in adults. ORP-011 is currently under non-clinical development.