Pediatric medicines have long been neglected by the pharmaceutical industry, who mainly focused their efforts on adult medicines. There are reasons for this situation: the maturing organism of children poses specific and significant challenges with regards to clinical trials and pharmaceutical development and the market is often too small to attract major pharma consideration.
Consequently, there has been significant “off label” use of adult medicines in the pediatric population. Dosage forms designed for adults have often been modified for administration to children (e.g. segmenting tablets, cutting transdermal patches), thus creating a risk for misuse or overdosage.
At ORPHELIA Pharma, we believe that developing new 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 in bringing new medicinal products to the market in niche indications where the medical need is the most salient: in neuro-pediatry (epilepsy, neuro-degenerative diseases) and in metabolic diseases.
The current company set-up is focused on product development and commercialization.
We are actively seeking partners that share our vision and commitment to children care.
Our commitment to improve the health condition of severely debilitating childhood diseases is deeply rooted in our history and in that of Hugues Bienaymé, the founder of the company.
In August 1968, Hugues’ father, Pr. Jean Bienaymé, then principal surgeon at Enfants Malades Hospital (Paris), successfully operated and separated two Togolese Siamese sisters, and this was a breakthrough achievement at that time.
During most of his academic career, as Head of Saint Vincent-de-Paul Hospital pediatric unit, he pioneered life-saving surgical treatments of several rare childhood pathologies, such as duodenum stress ulceration, childhood malformative pancreatitis, Curling’s ulcer.
In 2007, TargeOn SAS was incorporated in Paris by Hugues Bienaymé to pursue our commitment to children’s care and rare diseases through medical treatments.
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.
Since then, we have reinforced our project pipeline in CNS and metabolic diseases and continuously sought for new opportunities in these domains.
In 2015, TargeOn was renamed ORPHELIA Pharma to better reflect its commitment to children’s care. The company became a Société Anonyme, and a Board of Directors was set up.
ORPHELIA Pharma’s team is composed of seasoned experts in clinical research, drug discovery, 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 organisation lean and agile.
Hugues graduated from the Ecole Normale Supérieure de Saint-Cloud in 1986. After an Agrégation of physical sciences and chemistry, he gained a PhD in organic chemistry at the Paris XI University (under the supervision of Pr. A. Lubineau), followed by a postdoctoral stay at the University of Geneva with Pr. W. Oppolzer (total synthesis of natural alkaloids).
He joined the pharmaceutical industry in 1991 with Rhône-Poulenc (then Aventis, now Sanofi) where he became familiar with drug discovery through combinatorial and medicinal chemistry. After several rewarding R&D positions at Rhône-Poulenc/Aventis, he started his own venture by creating Chrysalon, a biotech company specialized in high-throughput drug discovery in 2001.
Hugues then joined Urogene, a biotech company specialized in urological diseases in 2002, as General Manager and CSO. There, he headed the preclinical development of several kinase inhibitors and GPCR modulators targeting prostate and bladder cancers. He also actively participated in the Phase II clinical development of besipirdine, an inhibitor of norepinephrine recapture, in stress urinary incontinence.
After selling the high-throughput discovery platform (Chrysalon) to Pierre Fabre Medicament in 2005, he took the head of the newly created subsidiary Pierre Fabre Urology and pursued the discovery and development of several kinase inhibitors to target urological and pediatric cancers.
In 2007, with the support of M. Pierre Fabre and Pierre Fabre Médicament, Hugues founded TargeOn SAS (now ORPHELIA Pharma SA).
Hugues has authored or co-authored 48 articles in peer-reviewed journals and 21 patents.
Gilles has 30 years of experience in the European pharmaceutical industry. After its PhD at Institut Gustave-Roussy, Villejuif, France, Gilles joined the Mérieux Group in 1986, then founded and managed IMTIX, then IMTIX-SangStat from 1994 to 2000, and eventually founded OPi in 2000, a company dedicated to rare and severe diseases. OPi was acquired by EUSA Pharma in 2007, now part of Jazz Pharmaceuticals.
Gilles is the President and Founder of Octalfa SAS, a family-owned, independent investment company specializing in Life Sciences.
Gilles is the founder or co-founder of several biotech or pharma companies in France and has developed multiple products and devices, at least 5 of them are on the market, approved almost worldwide.
Gilles was elected Executive Chairman of ORPHELIA Pharma in 2015.
Séverine Martin, MSc (Assistant Project Manager)
Séverine graduated from Montpellier University (MSc in organic chemistry) in 2004 and since then has gained a comprehensive professional experience in drug discovery for several companies, such as Servier, Proskelia (now Galapagos) and Cerep.
Séverine has been with ORPHELIA Pharma/TargeOn since its inception in 2007 and has been implemental in all discovery and development programs of the company.
André Tartar, PhD, PharmD (Member of the Board)
André is a founding member and a shareholder of ORPHELIA Pharma. His broad knowledge encompass organic chemistry, pharmaco-chemistry, peptides and proteins, molecular pharmacology and immunochemistry. André is Professor at the Lille Faculty of Pharmacy since 1981 and at Institut Pasteur of Lille since 1995. Besides his academic endeavors, he is or has been at the scientific board of several companies and institutions including Ferring, Trophos, CEREP, Paris Biotech Santé and LEEM Recherche. He is past president of the Société de Chimie Thérapeutique.
André has published over 240 articles in leading scientific publications and has authored over 20 patents.
Emmanuèle Gerdil, PharmD (Head of Regulatory Affairs, acting)
Emmanuèle graduated in Pharmacy from the University of Lyon, and after 15 years as Clinical Biologist at Hôpital Edouard Herriot, Lyon, Emmanuèle joined Sanofi Pasteur in 1990 as vaccines Quality Control Manager. In 1994 she was appointed Qualified Person and Regulatory Affairs Manager for Sanofi Pasteur MSD, the European joint-venture between Sanofi Pasteur and Merck & Co, leading companies in vaccines and biotechnology products. She was then promoted Executive Director, Regulatory Affairs Europe at Sanofi Pasteur MSD.
Emmanuèle was 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 Attali 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). Pierre is advising ORPHELIA Pharma on its overall clinical strategy and is involved in the preparation of the clinical protocols.
Pierre has contributed to the approval of more than 10 new drugs and has authored or co-authored more than 100 articles in peer-reviewed journals and patents.
Gérard Damien, Chem Eng. (CMC and Formulation)
Gérard graduated from the Institut National des Sciences Appliquées de Lyon. He was successively Head of analytical development department at Servier, then Associate Director of the pharmaceutical development center, Groupe de Recherche Servier and Director, Head of pharmaceutical development, Groupe de Recherche Servier. He is involved in many quality and regulatory bodies, such as the French Pharmacopoeia, the European Pharmacopoeia and EFPIA.
Gérard is advising ORPHELIA Pharma on its overall CMC and formulation strategy and is also advising on quality aspects.
Shareholders of ORPHELIA Pharma
Pierre Fabre is the 3rd largest French pharmaceutical group and the 2nd largest dermo-cosmetics laboratory in the world. In 2014, its sales reached €2.1 Billion, with revenues in dermo-cosmetics accounting for 55% and international sales for 55%. Founded and its headquarters still based in the South-west of France, Pierre Fabre currently has branches in 44 countries and distribution agreements in over 130 countries. Covering a continuum of healthcare products, from prescription drugs and consumer health care products (family care, oral care, natural health) to dermo-cosmetics, Pierre Fabre Laboratories employ over 10,000 people worldwide. In 2014, Pierre Fabre dedicated more than 17% of its drug revenues to R&D. www.pierre-fabre.com
Founded by Gilles Alberici, initiative OCTALFA is a family-owned organization based in France comprising investment companies and private foundations. Its two investment companies OCTALFA and OTONNALE are based on entrepreneurship spirit applied to life sciences and sustained development, respectively. Since 2007, initiative OCTALFA has supported 14 innovative companies in France, with up to 2.5 m€ funding per company. It also seeded OCTALFA 360 enjeux d’avenir (a mutual fund specialized in life sciences, energy and cleantech) and is a shareholder of R2V, a French venture fund investing in the South-East of France. In addition, and through the Dominique & Tom Alberici Foundation, more than 60 charity projects have been financed by initiative OCTALFA since 2008 to improve well-being of disabled people and cancer patients in France and in Madagascar. www.initiative-octalfa.eu
Board of Directors
Gilles Alberici, Chairman of the Board
Hugues Bienaymé, Member of the Board
André Tartar, Member of the Board
Frédéric Desdouits, Observer (on behalf of Laboratoires Pierre-Fabre)
The Board of Directors will be strengthened in the coming months in order to support development activities aiming at building international partnerships.
At ORPHELIA Pharma, we highly welcome external innovation. Over the years, we have built strong collaborative relationships with several leading academic institutions.
What is a PUMA?
PUMA stands for Pediatric Use Marketing Authorization. It was set-up in 2007 by the European Parliament and Council to encourage the development of drugs adapted for pediatric patients.
A pediatric drug is deemed eligible to be reviewed by the European Medicines Agency under the “PUMA status” when it obtains approval on its Pediatric Investigation Plan (PIP). This plan is approved after a thorough review of the submitted file and defines the development plan that is deemed necessary to obtain the Marketing Authorization in the European Union.
More information on PUMA can be obtained at the EMA website.
PUMAs have now been tested and implemented on some products but only very few companies seem to have been structured to focus on this opportunity. ORPHELIA Pharma’s ambition is to leverage PUMAs and Orphan Drug Designation status and quickly become a European leader in pediatric neurology, developing and marketing a portfolio of products in this specialty market.
Why ORPHELIA Pharma ?
ORPHELIA Pharma was incorporated in 2007 as TargeOn. The company’s name was subsequently changed to ORPHELIA Pharma to better reflect our commitment to children’s care.
The name ORPHELIA Pharma was chosen in tribute to the Greek legendary hero Orpheus. Since our childhood, myths fill our world with inspiring and powerful symbols. The name 'Orpheus' resonates in everyone's imagination to conjure up a world of dream and possibility. Legend has it that Orpheus was given a lyre by his father Apollo. Orpheus transformed his lyre into a unique instrument able to charm humans, animals and even rocks. By perfecting his lyre, Orpheus was able to infuse hope and joy around him.
At the height of his art, Orpheus succeeded in bringing back Eurydice from the Inferno (a sick condition) to the surface of earth (a healthy state).
We found inspiration in this metaphor which embodies our mission: developing new medicines to treat and improve the health of sick children and the quality of life of children and their family.