Our Vision

Pediatric medicines have long been neglected by the pharmaceutical industry. Children differ from adults in their biology, their needs for specific routes of drug administration and taste-masked medicines as well as the drug-related toxicities. It is therefore essential that drugs used to treat children are developed properly and using child-friendly formulations.

Many medicines used to treat pediatric patients lack such child-adapted forms. Consequently, adult medicines are manipulated by pharmacists, caregivers or the parents, who are forced to crush tablets, open capsules or split sachets and disperse into the drink or food, thus creating a risk for misuse and under or over dosage. Furthermore, many drugs have not been adequately developed in pediatric indications and there is a significant “off label” use of unlicensed adult medicines in children.

At ORPHELIA Pharma, we believe that developing pediatric drugs is not doomed to fail and could be successfully undertaken with the proper methodological approach and the ability to tap into the expertise of key partners. Commitment to children’s care is all we need to succeed.

We focus our efforts on developing and bringing new medicinal products to the market in orphan indications where the medical need is the most salient: neuropediatry (severe forms of epilepsy) and pediatric oncology conditions. All our development programs encompass a particular focus on the pharmaceutical formulation to ensure high quality, greater dose flexibility, easier administration and better acceptance of drugs by the pediatric patients.

Our mission is to significantly improve essential medicines administered to children.

Our History

ORPHELIA Pharma was first incorporated in 2007 as TargeOn by Hugues Bienaymé. In the early years of the company, several potent and selective kinase inhibitors were discovered and optimized to target rare forms of childhood leukemia.

In 2009, the company decided to expand its operations to orphan indications in the Central Nervous System (CNS) arena. The company commenced several collaborations with academic teams to find new cures to rare forms of epilepsy and to Rett syndrome, a neurodegenerative condition for which no treatments are available.

In 2010, pursuant to the new European regulation on pediatric medicines, the company embarked on the development of a pediatric form of vigabatrin, a potent antiepileptic drug with a unique mechanism of action.

In 2015, TargeOn was renamed ORPHELIA Pharma to better reflect its commitment to children’s care. Since then, we have reinforced our project pipeline in the fields of neurological diseases and pediatric oncology and continuously sought for new opportunities in these domains.

Our Team

ORPHELIA Pharma’s team is composed of seasoned experts in drug discovery, clinical research, development and regulatory activities. We have developed a unique corporate model with experts in different fields that contribute to ORPHELIA Pharma and its product development as needed over time. Over the years, we have also developed close collaborations with several world-leading academic clinical and research teams. This allows ORPHELIA Pharma to benefit from very experienced specialists, and the best research experts in our field, while keeping the organization lean and agile. ORPHELIA pharma also relies on a strong network of industrial collaborators.

  • Gilles Alberici, PharmD, PhD
    Chairman
    Gilles has 30 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 President and Founder of initiative OCTALFA, an organization dedicated to the inception and management of drug development companies. He was elected Executive Chairman of ORPHELIA Pharma in 2015.

  • Hugues Bienaymé, PhD
    General Manager and Chief Scientific Officer
    Hugues has more than 25 years of experience in pharmaceutical industry both in pharma (Aventis, Pierre Fabre) and in biotech companies (Chrysalon, Urogene). In 2007, he created TargeOn, a company focussed in the development of paediatrics medicines. TargeOn became ORPHELIA Pharma in 2015.

  • Jérémy Bastid, PharmD, PhD
    Chief Development Officer
    Jeremy joined ORPHELIA Pharma in 2015 as Chief Development Officer. He is a pharmacist and oncologist, who specialises in immuno-oncology. He is also R&D Director of initiative OCTALFA, an organization dedicated to the inception and management of drug development companies and Chief Operating Officer of OREGA biotech.

  • Virginie Beaurent, PharmD
    Regulatory Affairs Director
    Virginie joined ORPHELIA Pharma in 2016 as head of Regulatory Affairs and oversees the company’s regulatory filings. She has more than 10 years of experience in Regulatory Affairs both for France and international activities.

  • Mathieu Schmitt, PharmD, MSc
    Pharmaceutical Development (CMC) Director
    Mathieu joined ORPHELIA Pharma in 2017 and oversees all CMC activities with ours subcontractors. He has 10 years of experience in pharmaceuticals companies as head of the formulation laboratory, senior CMC development manager and was in charge of all CMC activities, from preclinical to clinical.

  • Séverine Martin, MSc
    Project Manager
    Séverine joined ORPHELIA Pharma/TargeOn since its inception in 2007. She is responsible of the coordination of Orphelia’s projects and manage the relationship with partners and subcontractors.

  • Claire Okaïs, Pharm D, MSc
    Medical and Scientific Affairs Director

    Claire joined ORPHELIA Pharma in 2018 and oversees medical and scientific affairs activities. She has 10 years of experience in pharmaceutical companies as an epidemiologist and medical Advisor for french and international activities in various therapeutic areas.

  • Emmanuèle Gerdil, PharmD
    Regulatory Affairs, Acting
    Emmanuèle joined ORPHELIA Pharma in 2015 as Regulatory Affairs consultant. She was Qualified Person, Executive Director, Regulatory Affairs Europe at Sanofi Pasteur MSD during 15 years. Emmanuèle was also an active member of the Regulatory Affairs Working Group of the European Vaccine Manufacturers (EVM), a specialized group of the European Federation of Pharmaceutical Industry Associations (EFPIA).

  • Pierre Attali, MD, MSc
    Chief Medical Officer, Acting
    Pierre is advising ORPHELIA Pharma on its overall clinical strategy and is involved in the preparation of the clinical protocols. He is a hepatologist with a long-lasting commitment to clinical development. He was involved in the development of multiple products during his career at Synthelabo (now Sanofi) as well as at several biotech companies (Urogene, Bioalliance Pharma/Onxeo, Sensorion, Selexel).

Shareholders of ORPHELIA Pharma

Pierre Fabre
Pierre Fabre is the 2nd largest private French pharmaceutical group and the 2nd largest dermocosmetics laboratory in the world. In 2016, Pierre Fabre generated 2,282 million euros in revenues, of which 60% came from its international business and 59% from its dermo-cosmetics division. Headquartered in the South-West of France, Pierre Fabre counts more than 13,000 employees worldwide, owns subsidiaries and offices in 47 countries and enjoys distribution agreements in over 130 countries. In 2016, Pierre Fabre dedicated ca. 195 million euros to its R&D efforts, split between oncology, central nervous system, consumer healthcare, dermatology and dermocosmetics.
For more information, please visit www.pierre-fabre.com

Initiative OCTALFA
Founded by Gilles Alberici, initiative OCTALFA is a French, self-funded and independent organization dedicated to building companies in the healthcare field. IO was founded on the ground of our previous company, OPi, Pharmaceuticals for Rare Diseases, incepted in 1999 and now part of Jazz Pharmaceuticals. Since 2007, we participated or setup 14 companies. IO focuses its R&D efforts on the development of therapies for orphan indications, notably for the pediatric population. We strive on projects with high unmet needs in the field of oncology and hematology, neurology and metabolic disorders. Our objectives are to identify inventions (from hospital, academia or industry), coordinate research and feasibility studies prior to the inception of a dedicated company, define its strategy, set up the management and fund activities until the market introduction of products or structuring partnerships. As a side note, IO is also housing a Foundation dedicated to cancer patients and disabled people.
For more information, please visit initiative-octalfa.eu

Board of Directors

Gilles Alberici, Chairman of the Board
Hugues Bienaymé, Member of the Board
Pierre Attali, Member of the Board
Julien Alberici, Member of the Board (on behalf of Initiative Octalfa)
Maurice Chelli, Observer (on behalf of Pierre Fabre)