Committed to the development of essential medicines for rare pediatric diseases.
ORPHELIA Pharma is a French pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric orphan diseases.
ORPHELIA Pharma‘s mission is to provide patients with essential products in the fields of rare diseases, with formulations adapted to the pediatric population.
With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.
Our first product, Kigabeq®, received EU marketing authorization in 2018 for the treatment of West syndrome (infantile spasms) as monotherapy and for the treatment of resistant partial epilepsy (focal onset seizures) in combination with other antiepileptic medicinal products, and is now available in several countries.
Kizfizo®, an oral liquid formulation of temozolomide, is currently under EU marketing authorization review for the treatment of relapsed or refractory neuroblastoma. It is distributed under the name Kimozo through an early access program in France since the 1st of June 2022 and an expanded access program in Europe.
Committed to the development of essential pediatric medicines.
ORPHELIA Pharma’s team is highly experienced and ambitious, with large experience and proven track record of successfully identifying, developing and commercializing medicines that advance patient care and improve their quality of life in rare pediatric medicines.
Over the years, we have built collaborations with world-leading academic clinical and research teams. This allows ORPHELIA Pharma to benefit from very experienced specialists, while keeping the organization lean and agile. ORPHELIA Pharma also relies on a strong network of industrial collaborators.
Gilles has more than 35 years of experience in the pharmaceutical and biotechnology sectors. He created and developed OPi, Pharmaceuticals for Rare Diseases and has taken more than ten products through clinical trials. Gilles is the Founder and CEO of initiative OCTALFA.
General Manager and Chief Scientific Officer
Hugues has more than 25 years of experience in pharmaceutical (Aventis, Pierre Fabre) and biotech (Chrysalon, Urogene) companies. In 2007, he created TargeOn, a company focused on the development of pediatric medicines. TargeOn became ORPHELIA Pharma in 2015.
Chief Pharmaceutical Affairs Officer
Laurent joined ORPHELIA Pharma in 2020 as Chief Pharmaceutical Affairs Officer. He is also ORPHELIA Pharma's Pharmacien Responsable (Qualified Person). Pharmacist and scientist by background, Laurent has more than 25 years of experience in successful drug development and registration approval, especially in rare and/or pediatric pathologies (Orphan Europe now Recordati Rare Diseases, DBV Technologies).
Chief Development Officer
Jeremy joined ORPHELIA Pharma in 2015 as Chief Development Officer. He is a pharmacist and oncologist, specialized in pediatric and immuno-oncology. Jeremy is also the CEO of OREGA Biotech and the R&D Director of initiative OCTALFA.
Chief Pharmaceutical Development Officer
Caroline joined ORPHELIA Pharma in 2019. Caroline has a 20-year career in pharma and biotech companies, including Onxeo (formerly Bioalliance Pharma) in which she contributed to the development, registration and marketing of several oncology drugs, and Stallergenes Greer.
Project Management and Supply Chain Manager
Séverine is at ORPHELIA Pharma since its inception in 2007. She is responsible for the coordination of ORPHELIA’s projects and manages the supply chain of ORPHELIA’s Products.
Pharmaceutical development / CMC Manager
Nawel joined ORPHELIA Pharma in December 2022 as pharmaceutical development / CMC Manager. She has more than 10 years of experience in the pharmaceutical industry and more specifically in industrial development (Laboratoires Guerbet). She has conducted several successful industrial transfers.
Quality Assurance Manager
Sarah joined ORPHELIA Pharma in 2020. She is pharmacist specialized in quality assurance. After a first experience for an international laboratory, she joined ORPHELIA Pharma to supervise its Quality Management System (QMS).
Pierre advises ORPHELIA Pharma on its clinical strategy and protocols. He is a hepatologist with a long-lasting commitment to clinical development. He developed multiple products at Synthelabo (now Sanofi) and at several biotech companies (Urogene, Bioalliance Pharma/Onxeo, Sensorion, Selexel).
Regulatory Affairs Advisor
Emmanuèle advises ORPHELIA Pharma as Regulatory Affairs consultant since 2015. She was Qualified Person, Executive Director, Regulatory Affairs Europe at Sanofi Pasteur MSD during 15 years and also active member of the Regulatory Affairs Working Group of the European Vaccine Manufacturers.
“In 2015, OCTALFA was considering founding a pharmaceutical company to develop medicines for rare pediatric diseases, like we did with OPi Pharmaceuticals for rare diseases at the beginning of the 2000s. OPi made it possible to market several essential drugs in oncology and neurology worldwide. When discussing with Hugues Bienaymé, we realized that we had common values and that we could use our resources to develop Orphelia Pharma.”
– Giles Alberici, CEO initiative Octalfa and Executive Chairman, Orphelia Pharma.