Discover ORPHELIA Pharma
Committed to the development of essential medicines for rare and pediatric diseases.
ORPHELIA Pharma is a French pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and 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 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.
Our second product, Ivozall®, obtained EU marketing authorization in 2019 for the treatment of relapsed acute lymphoblastic leukemia.
Kimozo®, an oral liquid formulation of temozolomide, is currently under clinical development for the treatment of relapsed or refractory neuroblastoma. Kimozo® is distributed through an early access program in France since the 1st of June 2022 and an expanded access program in Europe.
Hugues Bienaymé, founder of ORPHELIA Pharma states: “From my discussions with pediatricians, I became convinced that the needs for pediatric medicines were poorly met. In particular, the development of medicinal products adapted to the pediatric population had not been of interest for the pharmaceutical industry. The 2006 European Regulation on medicinal products for pediatric use pushed me to steer the development of ORPHELIA Pharma in this direction “.
“We collaborated with clinicians from the Necker-Enfants-Malades Hospital in Paris to develop a pediatric form of vigabatrin, an essential anti-epileptic drug for the treatment of children suffering from West syndrome. This work led to the European Marketing Authorization of Kigabeq®, an innovative formulation specifically designed for young children. It demonstrated that it is possible to successfully develop an essential drug for a rare and severe disease.
rare and serious diseases
Gilles Alberici, President of ORPHELIA Pharma highlights: “The regulation on pediatric medicinal products complements the regulation on orphan medicinal products adopted in 2000 by the European Union, which laid down a single European orphan designation procedure.
By means of various incentive measures, it encourages pharmaceutical companies to carry out research and development studies on orphan medicines and to market them. Medicines for purely pediatric indications often meet the criteria for orphan drugs and these two regulations have proven their effectiveness”.
Another therapeutic area of research at ORPHELIA is pediatric oncology. Leukemias, brain tumors and neuroblastoma are the main cancerous pathologies encountered in children under 15.
“At ORPHELIA Pharma, we focus on relapsed or refractory diseases for which the prognosis remains dismal. We collaborate with clinical pediatricians on indications with high unmet medical needs and for which we believe we can bring a technical or scientific solution to overcome it”, concludes Jérémy Bastid, Chief Development Officer of ORPHELIA Pharma.
“Alongside the clinical development of our products we also focus our efforts on developing a formulation adapted to the use in young children”.
in relapsed pediatric cancers
Since 2018, ORPHELIA Pharma has been working in close collaboration with Gustave Roussy Hospital, one of the largest cancer centers in Europe, on developing a liquid oral formulation of temozolomide for neuroblastoma, a rare cancer affecting very young children.
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 both in rare and 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, especially in rare 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.
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.
Project Manager 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.
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).
Regulatory Affairs, Acting
Emmanuèle advises ORPHELIA Pharma since 2015 as Regulatory Affairs consultant. 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.
Pierre is advising 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).
From its inception, the company has worked closely with Pierre Fabre Médicament, its historical shareholder. Since 2015, ORPHELIA Pharma has been financially supported by Initiative OCTALFA, the holding company of the Alberici family.
Gilles Alberici: “In 2015, OCTALFA was considering founding a pharmaceutical company to develop medicines for rare pediatric diseases, on the model of what we had done with OPi Pharmaceuticals for rare diseases at the beginning of the 2000s. OPi made it possible to market worldwide several essential drugs in oncology and neurology. When discussing with Hugues Bienaymé, we realized that we had common values and that we could use our resources to develop ORPHELIA Pharma ”.
At the end of 2019, three French family offices created by seasoned entrepreneurs joined the shareholding structure of ORPHELIA Pharma: Ravenala Holding (Alain Tornier), PAF Kapital (Jean-François Auffret) and CEMAG Care (André Ulmann).