Founded in 2007 by Hugues Bienaymé and previously known as TargeOn SAS, ORPHELIA Pharma’s mission is to develop and market new pediatric formulations of drugs already approved in Europe or in the USA. The company intends to use the regulation on orphan and on pediatric medicines, including notably the PUMA (Pediatric Use Marketing Authorization) regulation. ORPHELIA Pharma holds several projects with anti-epileptic and oncology drugs. The most advanced project is currently under European centralized marketing autorization review.


Kigabeq® is a reformulation of an existing antiepileptic drug, vigabatrin, which makes its administration to young patients easier and safer too. The clinical evaluation is completed and Kigabeq® is currently under EMA approval review.

Launched in 1989, vigabatrin (Sabril™) is the only available drug that acts by inhibiting the GABA transaminase enzyme. The product has been long marketed by Sanofi in Europe and since 2009 by Lundbeck in the USA. In 1999, the EMA re-evaluated the risk benefit ratio of vigabatrin and the product is now recommended in Europe for:
> Infantile spasms (severe epilepsy), a rare and devastating form of epileptic encephalopathy with peak incidence at about 6 months of age. It is a rare disease with about 2500 new cases/year in Europe. The prognosis is poor with deleterious effect on brain maturation and cognitive development, if left untreated.
> Pharmaco-resistant partial seizures.

Unmet medical need in pediatric patients

Vigabatrin is the first-line treatment for infantile spasms but current formulations are not adapted for pediatric use. 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”.

ORPHELIA Pharma has developed a solution to conveniently dose vigabatrin:
> Easy dosing by increments of 50 mg: one scored tablet of 100 mg and one scored tablet of 500 mg.
> Bioequivalent to Sabril™ sachet.
> Soluble tablets with fast water dispersion (< 30 seconds).
> Concentrated solutions can be obtained.
> Easy manipulation for care givers.
> Less wastage.

ORP-005, ORP-008 & ORP-009

ORP-005 is a paediatric formulation of an essential medicine used to treat CNS cancers. Due to the toxicity and bitterness of the drug substance manipulation and administration of the marketed adult medicine is difficult in the paediatric population. ORP-005 is a taste-masked formulation coupled with a dispensing device made to improve administration and compliance in infants and children. ORP-005 is currently under clinical evaluation.

ORP-008 is a drug targeting childhood neuroblastoma with a novel mechanism of action. ORP-008 is currently under preclinical development in this orphan indication.

ORP-009 ORP-009 is a drug that targets various CNS cancers by repositioning a known medicine. The dosage form developed is a new enteral/parenteral formulation which could be used for neonates, children and adults.