4 May 2021

Inclusion of the first patient in the TEMOkids pediatric clinical trial
4 May 2021

ORPHELIA Pharma announces the inclusion of the first patient in the TEMOkids pediatric clinical trial Paris and Lyon, on May 4th, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, announces the inclusion of a first patient in the TEMOkids pediatric clinical study. The TEMOkids study aims to determine the pharmacokinetic parameters, tolerance, acceptability and response to treatment of Kimozo®, the first oral suspension of temozolomide, in the pediatric population (www.temokids.eu). Temozolomide is an anti-cancer drug used in treatment regimens for refractory or relapsed neuroblastoma that mainly affect very young children. It is also used to treat rhabdomyosarcoma and certain brain tumors. The only oral forms of temozolomide currently available are capsules which are not suited to children. In fact, to be administered, the capsules must be opened and their content added to food. In young children, the formulation is essential to ensure correct medication intake, safety, accuracy of the delivered dose and adherence to treatment. To meet the requirements of onco-pediatricians and the needs of children, ORPHELIA Pharma, working in close collaboration with Gustave Roussy hospital teams, has developed Kimozo®, the first ready-to-use drinkable suspension of temozolomide. “The TEMOkids trial, during which our oral suspension of temozolomide will be administered to children for the first time, is a key stage in the clinical development of Kimozo®,” says Caroline Lemarchand, Chief Pharmaceutical Development Officer for ORPHELIA Pharma, “This study will evaluate the pharmacokinetics of temozolomide, alone or in combination with other treatments, in children from 1 year of age. It will also define the tolerance and acceptability of Kimozo® within the study population, and collect data on tumor response to treatment. The first results of this clinical trial are expected mid-2022.“ “We are very pleased to be involved in the clinical development of this long-awaited new pediatric formulation of temozolomide for the treatment of cancer in children,” says Dr Samuel Abbou, pediatric oncologist at the Gustave Roussy hospital, and coordinating investigator of the TEMOkids study. “The study plans to recruit 40 children in 5 countries; that is France, Germany, Spain, the UK and the Netherlands, thanks to the support of a structured European-scale pediatric oncology network.” “We have received an enthusiastic response from the members of the European pediatric oncology community who have chosen to participate in this clinical trial,” says Hugues Bienaymé, General Manager and Chief Scientific Officer for ORPHELIA […]
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26 April 2021

ORPHELIA Pharma recrute un/une Assistant(e) de direction, Office Manager
26 April 2021

Basée à Paris et à Lyon, ORPHELIA Pharma SAS est une société biopharmaceutique dédiée au développement et à la commercialisation en Europe de médicaments pédiatriques en oncologie et en neurologie. Sa mission est d’améliorer significativement les médicaments administrés aux enfants en développant des produits efficaces, sûrs et faciles à utiliser. ORPHELIA Pharma conduit des projets de recherche innovants à travers des collaborations académiques et industrielles. ORPHELIA Pharma possède plusieurs projets dans le domaine de la neurologie et de l’oncologie. Ses deux produits les plus avancés, Kigabeq® et Ivozall®, sont actuellement en phase de lancement commercial. Un autre médicament est en développement clinique et pourrait obtenir un statut d’exception (de type ATU de cohorte) en 2021. Vous disposez d’une solide connaissance de la gestion administrative et vous êtes familier avec le rôle d’Office manager et de la relation avec les fournisseurs, les clients et les sous-traitants. Vous maitrisez parfaitement l’outil informatique (suite Microsoft). Vous êtes reconnu(e) pour votre sens de l’organisation, votre esprit d’initiative, votre communication aussi bien orale qu’écrite et vous avez un goût prononcé pour la qualité et le respect des délais. Vous disposez d’un excellent relationnel. Une bonne maîtrise de l’anglais parlé et écrit est indispensable. Vous avez envie de participer au lancement de produits essentiels en neurologie et en oncologie pédiatrique. Au sein d’ORPHELIA Pharma, vous serez en charge: De la gestion administrative du site – Services Généraux: organisation des bureaux, permanence téléphonique, accueil des visiteurs, gestion du parc informatique, support aux collaborateurs ; Du respect de certaines tâches liées à l’établissement pharmaceutique: responsabilité des archives internalisées et/ou externalisées, coordination des réponses aux appels d’offre pour les marchés hospitaliers, suivi des contrats et des partenaires de distribution ; Du suivi administratif et commercial dans le cadre d’un programme ATU: point de contact pour le programme, coordination des partenaires. Type de contrat : CDI temps plein. Poste basé à Paris. H/F Rémunération en fonction de l’expérience. Vous pouvez adresser votre candidature à l’adresse : contact@orphelia-pharma.eu 
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6 April 2021

License agreement for the distribution of Kigabeq® in 14 european countries
6 April 2021

ORPHELIA Pharma and Biocodex sign a license agreement for the distribution of Kigabeq® in 14 european countries Paris and Gentilly – April 6th 2021 – ORPHELIA Pharma and Biocodex announce today the execution of an exclusive distribution and marketing agreement for Kigabeq® (vigabatrin) for most of the European Union territory, including France. Kigabeq®, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq® is approved in Europe, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA). “We are very pleased to release this collaboration agreement with Biocodex“, says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, “With their long-standing experience in the field of pediatric epilepsy, Biocodex is certainly the best partner for Kigabeq® distribution in Europe“. “With Kigabeq®, the only pediatric presentation of vigabatrin, Biocodex is expanding its portfolio of drugs intended for rare and serious pathologies in children” adds Nicolas Coudurier, CEO of Biocodex, “ Kigabeq® and Diacomit® (stiripentol, developed and marketed by Biocodex) are two drugs which address unmet medical needs for young patients affected by severe and resistant to treatment epilepsies”. “Our objective is to make Kigabeq® available to all European clinicians, so that children affected by West syndrome can benefit.” concludes Gilles ALBERICI, President of ORPHELIA Pharma, “Thanks to this agreement with Biocodex, Kigabeq® will soon be prescribed in most of the European territory.” About Kigabeq® Kigabeq® is the first pediatric formulation of vigabatrin, an essential anti-epileptic drug. Presented as soluble and scored tablets of 100 mg and 500 mg for oral or nasogastric administration, Kigabeq® is notably indicated for the treatment of infantile spasms (West syndrome), an extremely serious epileptic encephalopathy of the infant. Kigabeq® was developed exclusively for children and has been granted European Pediatric Marketing Authorization (PUMA). About infantile spasms West syndrome, or infantile spasms, is an extremely serious epileptic encephalopathy in infants that combines epileptic spasms, psychomotor deterioration and a hypsarrhythmic type electroencephalogram. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in an infant with previously normal development or with a pre-existing delay; in all cases, infantile spasms severely hamper the psychomotor development. Pharmacological treatment should be started quickly to allow the spasms to stop and to improve the prognosis.  About Biocodex Funded in 1953 beyond the development and marketing of […]
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2 February 2021

Opening of the operating pharmaceutical establishment
2 February 2021

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.
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