CHMP has adopted a positive opinion for Kigabeq®

Paris and Lyon, August 28th, 2018 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology, announces today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the issuance of a marketing authorization for the medicinal product Kigabeq® by the European Commission. As Kigabeq® has been exclusively developed for use in children, it should receive a Paediatric Use Marketing Authorization (PUMA).

Kigabeq® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug and will be available as scored soluble tablets of 100 mg and 500 mg strengths. Kigabeq® is expected to be used as first-line treatment for infantile spasms, an extremely severe early childhood encephalopathic epilepsy.

« We are particularly honoured with this decision which comes after several years of continuous efforts of ORPHELIA Pharma together with our academic partners », comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « This positive opinion is a key milestone in the process to make Kigabeq®, our first medicinal product, available to the pediatric community ».

« The CHMP has acknowledged the interest of Kigabeq® in improving health of epileptic children. We are eagerly awaiting the final marketing authorisation from the European Commission by October », highlights Gilles Alberici, Chairman of ORPHELIA Pharma. « Kigabeq® is expected to be the fourth human medicine to be granted a PUMA in Europe ».

About infantile spasms

Infantile spasms, or West Syndrome, is an early childhood epileptic encephalopathy which combines axial spasms in cluster, a marked psychomotor delay and an hypsarythmic electroencephalogram. It is a rare disease with an estimated incidence of around 0.05 per 10,000. It affects infants who were previously normal or children with impaired cognitive development. Children with infantile spasms must be treated rapidly for spasms cessation and to improve prognosis.

About ORPHELIA Pharma

Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament.

Pierre Attali joins ORPHELIA Pharma Board Of Directors

Paris and Lyon, June 25th 2018 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces today that Pierre Attali has joined its Board of Directors.

Pierre Attali, MD, will bring to ORPHELIA Pharma his expertise in clinical development, especially in oncology, acquired during a 30-year career in pharmaceutical and biotech companies. Dr. Attali was successively Head of Clinical Research Department, Licensing Director and General Manager at Synthelabo, Chief Operating Officer and Chief Medical Officer at Bioalliance / Onxeo, Chairman and Chief Executive Officer at Urogene. More recently, he joined Sensorion as Chief Medical Officer and Head R&D. He also founded and managed several biotech companies, such including OSMO, Molecular Engines Laboratories (MEL), and more recently Selexel.

Pierre Attali contributed to the development, registration and marketing of more than 10 novel  drugs and formulations, both in Europe and in the United States. He authored numerous peer-reviewed scientific publications and patents.

« We are particularly honoured to welcome Pierre at ORPHELIA Pharma » comments Gilles Alberici, Chairman of ORPHELIA Pharma. « His deep knowledge of drug development, especially in the oncology therapeutic area, will be invaluable to us ».

 « I am very pleased to join the Board of ORPHELIA Pharma » says Pierre Attali; « I personally know and appreciate the management team since many years. The culture of the company, aiming at improving patient’s care and focusing on paediatric medicines is paramount to my own personal values».

About ORPHELIA Pharma

Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament.

ORPHELIA announces the filing of the Marketing Authorization for Kigabeq®

Paris and Lyon, February 27th 2018 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology announces today that it has submitted a European Centralized Marketing Authorization Application (MAA) for Kigabeq® in 2017.

Kigabeq® is the first pediatric formulation of vigabatrin, an essential antiepileptic drug, and will be available as scored soluble tablets of 100 mg and 500mg strengths. Kigabeq® is expected to be used as first-line treatment for infantile spasms (West syndrome), an extremely severe early childhood encephalopathy. Kigabeq has been exclusively developed for use in children and is eligible to a Pediatric Use Marketing Authorization (PUMA).

« We take great pride in having developed this unique formulation, which is also our first product», comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « It seemed to us that it was of paramount interest to propose a new formulation adapted to the pediatric population, which represents the vast majority of patients treated with vigabatrin in Europe».

« We supported the development of Kigabeq® from the beginning.» says Pr. Rima Nabbout, Head of the Pediatric Neurology Department at Necker – Enfants Malades Hospital. « The benefits of this new formulation rely in a better dose to body weight adjustment and a greater ease of use ».

An acceptability, tolerance and pharmacokinetic study was carried out in 24 French hospitals between 2014 and 2016, with 38 children suffering from infantile spasms or pharmaco-resistant focal epilepsy. The results of this study, presented at the congress of the French Pediatric Neurology Society (SFNP), which took place in Bordeaux between 31st January and 2nd February 2018, demonstrated a good adherence to treatment and an accurate dose to body weight adjustment.

About West Syndrome

West Syndrome, or infantile spasms, is an early childhood epileptic encephalopathy which combines axial spasms in cluster, a marked psychomotor delay and an hypsarythmic electroencephalogram. It is a rare disease with an estimated incidence of around 0.05 per 10,000. It affects infants who were previously normal or children with impaired cognitive development.

About ORPHELIA Pharma

Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament.

ORPHELIA PHARMA strengthens its capital base

Paris, January 30, 2018 ORPHELIA Pharma, a French biopharmaceutical dedicated to the development and marketing of pediatric drugs in the field of oncology and neurology today announces that it has strengthened its capital base in December 2017. The completion of this round of financing, subscribed exclusively by initiative OCTALFA, will allow the company to accelerate its projects with the ambitious objective to obtain three European marketing authorizations by the end of 2020.


Our program for the three years to come is particularly rich with the opening of our pharmaceutical facility, the launch of three products, the recruitment of key employees and the strengthening of our academic and industrial research collaborations“, comments Hugues Bienaymé, founder and General Manager of ORPHELIA Pharma. “Our activity is centered on the development of child-friendly formulations of essential drugs for the management of rare forms of epilepsy and for the treatment of the most aggressive forms of pediatric cancers for which the medical needs remain very high. Then, we will progressively add early-stage R&D projects, including the development of new molecules for these rare indications“.

ORPHELIA Pharma also announces that it has relocated its headquarters within the Quartier Latin in Paris and it has opened a branch in Lyon. ORPHELIA Pharma has also strengthened its team with the recruitment of Mathieu Schmitt, PharmD as CMC Director in charge of pharmaceutical development and Jeremy Bastid, PharmD, PhD who will be the Chief Development Officer of the company.


We are very proud to be part of the transformation of ORPHELIA Pharma, whose activity perfectly fits our values and expertise”, comments Gilles Alberici, President of initiative OCTALFA and ORPHELIA Pharma. We have been involved in ORPHELIA Pharma since 2015 and are delighted to see that the company has already achieved significant milestones in thirty months.

About ORPHELIA Pharma SAS

Based in Paris and Lyon, ORPHELIA Pharma SAS is a French biopharmaceutical company dedicated to the development and marketing of oncology and neurology pediatric drugs. Its mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. ORPHELIA Pharma also leads innovative R&D projects through academic and industrial partnerships. Key investors include initiative OCTALFA (lead investor) and Pierre Fabre Medicament. For more information, please visit www.orphelia-pharma.eu

ORPHELIA at the 2017 SIOP meeting in Washington DC

ORPHELIA Pharma will be present at the 49th Annual Congress of the International Society of Paediatric Oncology in Washington DC. http://www.siop2017.kenes.com/

Please meet us there (contact: jeremy.bastid@orphelia-pharma.eu)

ORPHELIA Pharma at AES 2017 in Washington DC

ORPHELIA Pharma will be present in Washington DC on December 1st to 4th, for the next Annual Epilepsy Society meeting.

From best practices to breakthrough thinking, the AES Annual Meeting offers the most extensive education on everything epilepsy. https://www.aesnet.org/