ORPHELIA Pharma and Gustave Roussy announce issuance of European Patent covering Kimozo®, the first oral liquid formulation of temozolomide

Paris and Lyon, on June 16, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs and Gustave Roussy, the leading cancer center in Europe, announce today that the European Patent Office has issued Patent EP3613436, which is directed to Kimozo®, the first pediatric, ready-to-use and taste-masked oral suspension of temozolomide. Kimozo® results from the collaboration between the pharmacists and clinicians from Gustave Roussy and ORPHELIA Pharma.

Temozolomide is an essential oncology drug used to treat relapsed or refractory neuroblastoma, a disease that unfortunately affects very young children. We are working in close collaboration with European clinicians and our goal is to make this pediatric form of temozolomide available as early as 2021 through early access programs”; commented Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma.

“We have developed a hospital compounded liquid preparation of temozolomide in 2015 to address the needs of young patients treated at our center”, explain Maxime Annereau, pharmacist and Samuel Abbou, pediatric oncologist at Gustave Roussy, both at the origin of this project. “This hospital formulation was suitable for children but did not exhibit the physico-chemical and stability properties compatible with an industrialized product. This product was therefore not available to all patients. Three years of research and development between Gustave Roussy and ORPHELIA Pharma were necessary in order to achieve the targeted specifications: a highly concentrated, taste-masked and stable formulation”.

“Our research efforts led to the development of Kimozo®, the first drinkable formulation of temozolomide. The innovation is based on the discovery of a key excipient involved in the stability and the control of the rheological properties of the suspension, an invention that has been patented”, concludes Jeremy Bastid.

The grant of Patent EP3613436 entitled “oral suspension of temozolomide” and co-owned by ORPHELIA Pharma and Gustave Roussy has been published in the European Patent Bulletin. Based on the filing date of the application, the patent protection is expected to last until 2038.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the pediatric population. Our first product, Kigabeq®, which was approved during the latter part of 2018 for the treatment of West Syndrome in particular, has been launched in several European countries. Our second product, Ivozall®, obtained a European marketing authorization at the end of 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma also conducts research projects through academic and industrial partnerships.

For further information, please go to www.orphelia-pharma.eu

Contact: Séverine Martin – +33 (0)1 42 77 08 18 – orphelia@orphelia-pharma.eu

About Gustave Roussy

Classed as the leading European Cancer Centre and the fifth on the world stage, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering with cancer. It is a source of diagnostic and therapeutic advances. It caters for almost 50,000 patients per year and its approach is one that integrates research, patient care and teaching. It is specialized in the treatment of rare cancers and complex tumors and it treats all cancers in patients of any age. Its care is personalized and combines the most advanced medical methods with an appreciation of the patient’s human requirements. In addition to the quality of treatment offered, the physical, psychological and social aspects of the patient’s life are respected. 3,200 health professionals work on its two campuses: Villejuif and Chevilly-Larue. Gustave Roussy brings together the skills which are essential for the highest quality research in oncology: a quarter of patients treated are included in clinical trials.

For further information: www.gustaveroussy.fr/en

Contact : Claire Parisel – +33(0)1 42 11 50 59 – +33(0)6 17 66 00 26 – claire.parisel@gustaveroussy.fr

About Kimozo®

Kimozo® (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address the needs of children. Kimozo® is an investigational medicinal product not yet approved for marketing.

16 June 2021

European Patent covering Kimozo®
16 June 2021

ORPHELIA Pharma and Gustave Roussy announce issuance of European Patent covering Kimozo®, the first oral liquid formulation of temozolomide Paris and Lyon, on June 16, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs and Gustave Roussy, the leading cancer center in Europe, announce today that the European Patent Office has issued Patent EP3613436, which is directed to Kimozo®, the first pediatric, ready-to-use and taste-masked oral suspension of temozolomide. Kimozo® results from the collaboration between the pharmacists and clinicians from Gustave Roussy and ORPHELIA Pharma. “Temozolomide is an essential oncology drug used to treat relapsed or refractory neuroblastoma, a disease that unfortunately affects very young children. We are working in close collaboration with European clinicians and our goal is to make this pediatric form of temozolomide available as early as 2021 through early access programs”; commented Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “We have developed a hospital compounded liquid preparation of temozolomide in 2015 to address the needs of young patients treated at our center”, explain Maxime Annereau, pharmacist and Samuel Abbou, pediatric oncologist at Gustave Roussy, both at the origin of this project. “This hospital formulation was suitable for children but did not exhibit the physico-chemical and stability properties compatible with an industrialized product. This product was therefore not available to all patients. Three years of research and development between Gustave Roussy and ORPHELIA Pharma were necessary in order to achieve the targeted specifications: a highly concentrated, taste-masked and stable formulation”. “Our research efforts led to the development of Kimozo®, the first drinkable formulation of temozolomide. The innovation is based on the discovery of a key excipient involved in the stability and the control of the rheological properties of the suspension, an invention that has been patented”, concludes Jeremy Bastid. The grant of Patent EP3613436 entitled “oral suspension of temozolomide” and co-owned by ORPHELIA Pharma and Gustave Roussy has been published in the European Patent Bulletin. Based on the filing date of the application, the patent protection is expected to last until 2038. About ORPHELIA Pharma ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the pediatric population. Our […]
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8 June 2021

ORPHELIA Pharma recrute un coordinateur d’étude clinique
8 June 2021

STAGE COORDINATEUR DE RECHERCHE CLINIQUE Basée à Paris et à Lyon, ORPHELIA Pharma SAS est une société biopharmaceutique spécialisée dans le développement et la commercialisation en Europe de médicaments pédiatriques et orphelins, plus particulièrement en oncologie et en neurologie. Sa mission est d’améliorer significativement la vie des patients 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. Ses deux produits les plus avancés, Kigabeq® et Ivozall®, sont actuellement commercialisés. Un autre médicament Kimozo® est en développement clinique. Pour en savoir plus sur notre laboratoire et nos activités, rendez-vous sur notre site internet : www.orphelia-pharma.eu Pour soutenir son programme de développement clinique, ORPHELIA recherche, pour son site basé à Paris, un stagiaire coordinateur d’étude clinique (h/f), étudiant (pharmacien 5ème année, master), pour une durée 6 mois et idéalement disponible immédiatement. En soutien au Directeur du Développement Pharmaceutique, en charge des opérations cliniques, vous assurez la gestion et le suivi des études cliniques en interaction avec les prestataires (CRO) impliqués. Vos activités principales seront les suivantes : Participation aux réunions d’avancement et aux visites (monitoring,…), Contribution à la rédaction des documents de l’étude, Organisation de la documentation des études cliniques, Revue des rapports de monitoring, Suivi des unités de traitements, Suivi et mise à jour des tableaux de bord de l’étude, Contribution à la rédaction et la revue des procédures d’assurance qualité. Qualifications / Compétences requises Stage entrant dans le cadre d’une formation de niveau Bac +5 scientifique avec une spécialisation en recherche clinique, Autonomie, rigueur, sens de l’organisation, méthodologie, communication, gout prononcé pour la qualité et le respect des délais, anglais scientifique, Respect des bonnes pratiques cliniques et de la réglementation en vigueur, Connaissance du déroulement de la recherche clinique, Maitrise des logiciels de bureautique. Merci d’adresser votre candidature par email à recrutement@orphelia-pharma.eu avec en objet « Stage Recherche Clinique ».
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20 May 2021

ORPHELIA Pharma recrute un/une Chargé(e) Qualité
20 May 2021

Apprentissage Chargé(e) Qualité ORPHELIA Pharma, laboratoire pharmaceutique en forte croissance recherche son/sa chargé(e) qualité, en contrat d’apprentissage, pour son établissement de Paris. Nous sommes spécialisés dans le développement et la commercialisation en Europe de médicaments pédiatriques et orphelins, plus particulièrement en oncologie et en neurologie. Notre mission est d’améliorer significativement la vie des patients en développant des produits efficaces, sûrs et faciles à utiliser. De taille humaine, notre équipe pluridisciplinaire maîtrise l’ensemble du cycle de vie des médicaments (développement pharmaceutique et clinique Europe / Etats-Unis, fabrication, exploitation France, commercialisation à l’international …). Ainsi, en rejoignant ORPHELIA Pharma vous aurez l’opportunité d’interagir en interne et en sous-traitance avec de nombreux métiers de l’industrie pharmaceutique, ce qui vous permettra de cerner rapidement les exigences et problématiques des différentes activités, mais également d’acquérir de solides compétences dans le fonctionnement d’un site exploitant. Vos missions : Au sein de l’équipe qualité et réglementaire, vous reporterez directement au Directeur des Affaires Pharmaceutiques / Pharmacien Responsable sur un projet important de l’entreprise : l’étude, la sélection et la mise en place d’une GED (Gestion Electronique des Documents) pour la création et la gestion des documents qualité du laboratoire. Vous serez en charge : • D’identifier et de synthétiser les besoins des collaborateurs • D’identifier, de sélectionner et de qualifier l’outil à mettre en place • De la mise en place de l’outil ainsi que de la formation des collaborateurs. Parallèlement à cette mission, vous apporterez votre support à l’équipe qualité et réglementaire sur les activités suivantes : • Participation à la rédaction de procédures et instructions • Aide au suivi des déviations, des CAPA et des Change Controls • Aide à la planification des audits et suivi des plans d’actions associés • Participation aux audits internes • Publication des avantages dans la base de transparence des liens. D’autres missions peuvent vous êtes confiées selon les besoins de l’entreprise et vos compétences.   Qualifications / compétences requises : • Formation de niveau Bac +5/+6 Ingénieur ou Pharmacien avec une spécialisation en Assurance Qualité • Autonomie, rigueur, curiosité, organisation, méthodologie • Qualité de rédaction et de synthèse écrite • Maitrise des outils bureautiques de base : Excel, Word, Powerpoint, Outlook • Capacité à comprendre et à rédiger des documents techniques en anglais. Contact : Le poste est à pourvoir pour septembre 2021 (à négocier selon les périodes de début d’alternance). Merci d’adresser votre candidature par email à recrutement@orphelia-pharma.eu avec […]
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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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