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 confi.

Kigabeq®

The first pediatric formulation of vigabatrin

Kigabeq® is a pediatric formulation of the antiepileptic drug vigabatrin

Kigabeq® makes the administration to young patients easier and safer. The clinical evaluation has been completed and Kigabeq® is currently under EMA approval review under a Pediatric Use Marketing Autorisation (PUMA) procedure.

Launched in 1989, vigabatrin (Sabril™) acts by inhibiting the GABA transaminase enzyme. The product has been marketed by Sanofi in Europe and since 2009 in the USA by Lundbeck. In 1999, the EMA re-evaluated the risk benefit ratio of vigabatrin and the product is now approved in Europe for the treatment of infantile spasms and pharmaco-resistant focal-onset seizures (partial seizures).

Infantile spasms are 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

Vigabatrin is the first-line treatment for infantile spasms but current formulations are not adapted for use in children. Vigabatrin is part of the “Revised priority list for studies into off-patent pediatric medicinal products” published by the EMA in 2014 with the need of an “age-appropriate formulation”.

Kigabeq® allows safe and convenient dosage of vigabatrin

  • Scored tablets of 100 mg and 500 mg, allowing 50 mg dosing steps
  • Bioequivalent to Sabril™ sachet
  • (Oro)dispersible tablets with fast water dispersion (< 30 seconds)
  • Possibility to obtain concentrated solutions
  • Easy manipulation for care givers
  • Reduced wastage

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.