ORPHELIA Pharma’s mission is to provide meaningful advances to children through the development of efficient, reliable and easy-to-use medicines. Our projects are focused on the development of novel pediatric formulations of approved drugs, approved drugs in new pediatric indications and new drugs in pediatric orphan or ultra-orphan indications. Our therapeutic areas of interest cover pediatric neurology (epilepsy) and rare pediatric oncology and hematology conditions.

Pipeline actualisé

Kigabeq®

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.

Products in development

Preclinical and clinical stage programs

ORP-010

ORP-010 is a drug used to treat a rare hematological malignancy. The development has been completed and ORP-010 is currently under EMA review.

ORP-005

ORP-005 is a child-adapted formulation of an essential medicine used to treat a rare childhood cancer. ORP-005 is currently under clinical evaluation.

ORP-009

ORP-009 is a known medicine repositioned to treat rare childhood cancer. ORP-009 is currently under preclinical evaluation.