Grant obtained from the region Île-de-France to support business activity development in Europe and the United States
26 January 2021

Opening of the operating pharmaceutical establishment

ORPHELIA Pharma opens its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France

Paris and Lyon, February 2nd, 2021. ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, announces today that it has opened its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France. According to the French Public Health Code (“Code de la Santé Publique”), ORPHELIA Pharma will now be able to commercialize medicines under its own name. The authorized activities include importation, sales, advertising, medical information, pharmacovigilance and batch monitoring. The establishment will also be able to launch and monitor early access programs (“Autorisation Temporaire d’Utilisation”, or ATU) on its own behalf or on behalf of third parties.

“As of today, we will be able to commercialize our own medicines in France. We currently have two pediatric medicines which have been approved in Europe through a centralized procedure: Kigabeq® (vigabatrin) and Ivozall® (clofarabine)”, comments Laurent Martin, Chief Pharmaceutical Officer of ORPHELIA Pharma. “Becoming a pharmaceutical establishment will also allow us to initiate an early access program (ATU) for our experimental medicine Kimozo®, a liquid oral formulation of temozolomide developed for the treatment of relapsed or refractory neuroblastoma, following validation by health authorities”, he adds.

After having obtained the authorization to open our pharmaceutical establishment from the French medicines agency (ANSM) on November 18th, 2020, this effective opening is a major milestone for our company and is a testament to the efforts undertaken by the people at ORPHELIA Pharma”, concludes Hugues Bienaymé, General Manager.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorization in September 2018 and has been made available in several countries. Our second product, Ivozall®, obtained European marketing authorization in November 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.