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# ORPHELIA Pharma

## Sitemaps
[XML Sitemap](https://www.orphelia-pharma.eu/sitemap_index.xml): Includes all crawlable and indexable pages.

## Posts
- [International Rare Disease Day 2026](https://www.orphelia-pharma.eu/news/international-rare-disease-day-2026/): On #InternationalRareDiseaseDay 2026, we are proud to join the global movement to recognise the 300 million people worldwide living with a rare disease.
- [Orphalan, a global pharmaceutical company specializing in the development and commercialization of orphan drugs, announces the acquisition of Orphelia Pharma, a French pharmaceutical company dedicated to rare and serious pediatric diseases in neurology and oncology.](https://www.orphelia-pharma.eu/news/orphalan-a-global-pharmaceutical-company-specializing-in-the-development-and-commercialization-of-orphan-drugs-announces-the-acquisition-of-orphelia-pharma-a-french-pharmaceutical-company-dedicated/): This strategic transaction further strengthens Orphalan’s position as a European leader with a global commercial footprint in rare diseases and expands our therapeutic portfolio.
- [Orphelia Pharma provides update on KIZFIZO® regulatory submission with the European Medicines Agency](https://www.orphelia-pharma.eu/news/orphelia-pharma-provides-update-on-kizfizo-regulatory-submission-with-the-european-medicines-agency/): Paris and Lyon, France, March 12th, 2025 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed on 27 February 2025 a negative opinion regarding the Marketing Authorization Application for KIZFIZO (temozolomide oral suspension) for the treatment of patients with relapsed or refractory high-risk neuroblastoma, further to the request for re-examination of the opinion submitted by the company in December 2024. The CHMP considered that the clinical benefit of KIZFIZO was not sufficiently demonstrated, preventing the establishment of a positive benefit-risk balance.
- [Orphelia Pharma provides update on the regulatory submission of KIZFIZO® with the European Medicines Agency](https://www.orphelia-pharma.eu/news/orphelia-pharma-provides-update-on-the-regulatory-submission-of-kizfizo-with-the-european-medicines-agency/): Paris and Lyon, France, November 18, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Marketing Authorization Application for KIZFIZO (temozolomide oral suspension) for the treatment of patients with relapsed or refractory high-risk neuroblastoma, because the CHMP considered that a positive benefit-risk balance has not been established at this stage. Orphelia will seek a re-examination of the opinion by the CHMP.
- [ORPHELIA Pharma to collaborate with Oscar Lambret clinical center to study KIZFIZO®, the first pediatric drinkable formulation of temozolomide, for the treatment of refractory or relapsed nephroblastoma (Wilms tumor)](https://www.orphelia-pharma.eu/news/orphelia-pharma-to-collaborate-with-oscar-lambret-clinical-center-to-study-kizfizo-the-first-pediatric-drinkable-formulation-of-temozolomide-for-the-treatment-of-refractory-or-relapsed-nephrob/): Paris and Lyon, June 11th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, is pleased to announce that KIZFIZO, temozolomide oral suspension, will be included in the MetroWILMS-1906 study (EudraCT: 2021-002540-67, NCT05384821). MetroWILMS is a French, multicentric, non-randomized, Phase 1-2 clinical trial to study the efficacy and safety of metronomic chemotherapy, including temozolomide, for the treatment of refractory or relapsed nephroblastoma (Wilms tumor). MetroWILMS is sponsored by the Oscar Lambret clinical center on behalf of the Société Française des Cancers de l’Enfant (SFCE), Nephroblastoma committee.
- [ORPHELIA Pharma recrute un/une Chef de Projet Réglementaire Senior / Senior Regulatory Project Manager](https://www.orphelia-pharma.eu/careers/orphelia-pharma-recrute-un-une-chef-de-projet-reglementaire-senior-senior-regulatory-project-manager/): 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 Kizfizo®, sont actuellement en phase de lancement commercial, ou en cours d’enregistrement et disponible en statut d’exception.
- [ORPHELIA Pharma and University of Birmingham strengthen their collaboration to support the registration of KIZFIZO®](https://www.orphelia-pharma.eu/news/orphelia-pharma-and-university-of-birmingham-strengthen-their-collaboration-to-support-the-registration-of-kizfizo/): May 22nd, 2024.  The University of Birmingham and Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of orphan paediatric medicines, are pleased to announce the extension of their collaboration to leverage patient-level data of the BEACON refractory or relapsed neuroblastoma trial to support the marketing authorisation of KIZFIZO.
- [Orphelia Pharma secures European patent covering KIGABEQ®,                                    the first pediatric formulation of vigabatrin](https://www.orphelia-pharma.eu/news/orphelia-pharma-secures-european-patent-covering-kigabeq-the-first-pediatric-formulation-of-vigabatrin/): Paris and Lyon, France, April 18th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, today announces that the European Patent Office (EPO) has issued an intention to grant patent number EP3426230 for KIGABEQ®.
- [International Drug Repurposing Conference 2024, 6-7th March 2024, Barcelona](https://www.orphelia-pharma.eu/news/international-drug-repurposing-conference-2024-6-7th-march-2024-barcelona/): The International Drug Repurposing Conference (#iDR24): Bridging Boundaries for Innovative Drug Repurposing, co-organized by REMEDi4ALL Consortium, Beacon for Rare Diseases, and MeRIT will be held on 6-7 March 2024 in Barcelona. 
- [ORPHELIA Pharma raises awareness for childhood cancer](https://www.orphelia-pharma.eu/news/orphelia-pharma-raises-awareness-for-childhood-cancer/): Today, February 15th, is the International Childhood Cancer Day. One out of 440 children is affected by cancer, i.e. 2 300 young patients every year in France. Although 80% of children are cured, one out of five with certain difficult indications does not survive. This ICCD is the opportunity for ORPHELIA Pharma to re-express its commitment to advancing care, treatments and research in the field of pediatric oncology.
- [ORPHELIA Pharma announces the publication of two scientific articles on KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma](https://www.orphelia-pharma.eu/news/orphelia-pharma-announces-the-publication-of-two-scientific-articles-on-kizfizo-the-first-pediatric-drinkable-formulation-of-temozolomide-for-the-treatment-of-refractory-or-relapsed-neuroblastoma/): Paris and Lyon, January 9th, 2024 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announces the publication of two scientific articles about KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma, which is under registration review under centralised procedure by the EMA.
- [ORPHELIA Pharma annonce la publication de deux articles scientifiques concernant KIZFIZO, première formulation pédiatrique buvable de témozolomide pour le traitement du neuroblastome réfractaire ou en rechute](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-la-publication-de-deux-articles-scientifiques-concernant-kizfizo-premiere-formulation-pediatrique-buvable-de-temozolomide-pour-le-traitement-du-neuroblastome-refractaire-ou-en/): Paris et Lyon, le 9 janvier 2024 – Orphelia Pharma, la société biopharmaceutique française dédiée au développement et à la commercialisation de médicaments pédiatriques dans les domaines de l'oncologie et de la neurologie, annonce la publication de deux articles scientifiques concernant KIZFIZO, première formulation pédiatrique buvable de témozolomide pour le traitement du neuroblastome réfractaire ou en rechute dont la demande d’autorisation de mise sur le marché est en cours d’évaluation par l’EMA à travers une procédure centralisée.
- [Orphelia Pharma secures US patent covering KIZFIZO®, first drinkable formulation of temozolomide](https://www.orphelia-pharma.eu/news/orphelia-pharma-secures-us-patent-covering-kizfizo-first-drinkable-formulation-of-temozolomide/): Company benefits from protection period until 2038
- [ORPHELIA Pharma to support the European paediatric Soft Tissue Sarcoma Study Group winter meeting.](https://www.orphelia-pharma.eu/news/orphelia-pharma-to-support-the-european-paediatric-soft-tissue-sarcoma-study-group-winter-meeting/): The European paediatric Soft Tissue Sarcoma Study Group (EpSSG; https://www.epssgassociation.it/) is holding its winter meeting on November 29th - December 1st, 2023, in Barcelona, Spain.
- [Medicines for Europe : 5th Value Added Medicines Conference, 7th November 2023, Brussels](https://www.orphelia-pharma.eu/news/medicines-for-europe-5th-value-added-medicines-conference-7th-november-2023-brussels/): The Medicines for Europe association represent the pharmaceutical companies supplying the largest share of medicines across Europe and is the voice of the generic, biosimilar and value-added industries.
- [Event : BioEurope Fall 2023 (November 6-8, 2023; Munich, Germany) ](https://www.orphelia-pharma.eu/news/event-bioeurope-fall-2023-november-6-8-2023-munich-germany/): Gilles ALBERICI, CEO at ORPHELIA Pharma and Hugues BIENAYME, General Manager will attend the BioEurope Fall meeting to be held November 6-8, 2023 in Munich, Germany. BioEurope convenes over 5,500 attendees, representing 60 countries and 2,220+ companies, making the event the industry's largest gathering of biopharma professionals in Europe.
- [Orphelia Pharma dépose une demande d’autorisation de mise sur le marché en Europe pour KIZFIZO®](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-depose-une-demande-dautorisation-de-mise-sur-le-marche-en-europe-pour-kizfizo/): Première formulation buvable de témozolomide pour le traitement des neuroblastomes réfractaires ou en rechute
- [Orphelia Pharma files EU marketing authorization application for KIZFIZO®](https://www.orphelia-pharma.eu/news/orphelia-pharma-files-eu-marketing-authorization-application-for-kizfizo/): Paris and Lyon, France, October 24, 2023 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, today announces the filing of a centralized Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for KIZFIZO®, the first oral liquid formulation of temozolomide.
- [ORPHELIA Pharma is at SIOP 2023 in Ottawa!](https://www.orphelia-pharma.eu/news/orphelia-pharma-is-at-siop-2023-in-ottawa/): Stop by our booth in the exhibition and poster area to learn more about our pipeline including KIZFIZO, temozolomide oral suspension (product in development).
- [Orphelia Pharma to present results of the TEMOkids study (pharmacokinetic,acceptability and safety of KIZFIZO®) at the 2023 SIOP annual meeting](https://www.orphelia-pharma.eu/news/orphelia-pharma-to-present-results-of-the-temokids-study-pharmacokinetic/): Paris and Lyon, France, October 4, 2023 – Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines announces today that results from the pediatric TEMOkids clinical trial will be presented at the 55th Société Internationale d’Oncologie Pédiatrique (SIOP) annual congress to be held in Ottawa, Canada, October 11-14, 2023. The results will be presented on October 14 during the oral session entitled “novel therapeutic approaches” by Dr. Lucy METAYER, co-investigator of the TEMOkids study and pediatric oncologist at Gustave Roussy, ranked 3rd cancer center worldwide (Villejuif, France).
- [Event : SIOPEN annual general meeting, October 4-6 2023, Ljubljana, Slovenia](https://www.orphelia-pharma.eu/news/event-siopen-annual-general-meeting-october-4-6-2023-ljubljana-slovenia-2/): Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma will attend the SIOPEN annual general meeting to be held on October 4-6, 2023 in Ljubljana, Slovenia. The SIOPEN annual meeting brings together about 200 clinicians, investigators, researchers and patient advocates from 34 European countries to discuss on-going clinical trials, results, and work in progress in the field of neuroblastoma. If you would like to arrange a meeting with Jeremy on October 5 or 6, please contact him at: jeremy.bastid@orphelia-pharma.eu
- [Event : ANR 2023 (15-18 May, Amsterdam, the Netherlands) ](https://www.orphelia-pharma.eu/news/event-anr-2023-15-18-may-amsterdam-the-netherlands/): Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma will attend the Advances in Neuroblastoma Research (ANR) meeting to be held May 15-18, 2023 in Amsterdam, the Netherlands. 
- [Event : SIOPe (May 8-12, Valencia, Spain)](https://www.orphelia-pharma.eu/news/event-siope-may-8-12-valencia-spain/): ORPHELIA Pharma will participate to the 4th Annual Meeting of the European Society for Paediatric Oncology (SIOPe 2023) to be held May 8-12, 2023 in Valencia, Spain. SIOPe aims to bring together the diverse stakeholders in a unique interactive format to discuss the current priorities and needs in the field of childhood cancers (https://siopeurope.eu/).
- [ORPHELIA Pharma selects Tanner Pharma to Initiate Named Patient Program for KIMOZO®](https://www.orphelia-pharma.eu/news/orphelia-pharma-selects-tanner-pharma-to-initiate-named-patient-program-for-kimozo/): Paris (France) and Charlotte, N.C. (USA), on April 18th, 2023 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, and Tanner Pharma Group, a global provider of integrated specialty access solutions, have signed an exclusive agreement to manage the supply and distribution of Ped-TMZ, also known as KIMOZO® (temozolomide 40 mg/ml, oral suspension), to meet the special demands which may come from European physicians for this unapproved pediatric form of temozolomide.
- [ORPHELIA Pharma announces completion of patient recruitment for the TEMOkids clinical trial](https://www.orphelia-pharma.eu/news/orphelia-pharma-announces-completion-of-patient-recruitmentfor-the-temokids-clinical-trial/): Paris and Lyon, March 2nd, 2023 – ORPHELIA Pharma, the French biopharmaceutical company that develops and markets pediatric medicines in the fields of oncology and neurology, announces the recruitment of the last patient of its TEMOkids clinical trial evaluating its ready-to-use temozolomide oral suspension, named KIMOZO® or Ped-TMZ, in children with a solid tumor from the age 1 year.The TEMOKids trial, during which KIMOZO® was administered to children for the first time, is a major milestone in its clinical development. In addition to generating pharmacokinetic data, the trial also evaluates the safety and acceptability of the suspension in the study population and collects data on treatment response.TEMOkids complements the study previously carried out in adults, which demonstrated the bioequivalence between KIMOZO® oral suspension and the capsule form of temozolomide, which is not adapted to children and yet regularly used in the absence of a pediatric form. ORPHELIA Pharma will present the results of the TEMOkids study at an upcoming scientific congress and will submit the marketing authorization dossier to the European Medicines Agency in 2023.
- [ORPHELIA Pharma annonce la fin du recrutement de son essai clinique TEMOkids](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-la-fin-du-recrutement-de-son-essai-clinique-temokids/): Paris et Lyon, le 2 mars 2023 – ORPHELIA Pharma, la société biopharmaceutique française qui développe et commercialise des médicaments pédiatriques dans les domaines de l'oncologie et de la neurologie, annonce le recrutement du dernier patient de son essai clinique TEMOkids évaluant la suspension buvable prête à l'emploi de témozolomide appelée KIMOZO® ou Ped-TMZ, chez l'enfant atteint d’une tumeur solide à partir de 1 an.L'essai TEMOKids, au cours duquel la suspension buvable de témozolomide a été administrée pour la première fois à des enfants, constitue une étape majeure de son développement clinique. Outre la génération de données pharmacocinétiques, l'essai évalue également la tolérance et l'acceptabilité de la suspension dans la population étudiée et collecte des données sur la réponse au traitement.TEMOkids complète l'étude précédemment menée chez l'adulte qui a démontré la bioéquivalence entre la suspension KIMOZO® et la forme gélule, non adaptée à l’enfant et pourtant régulièrement utilisée en pédiatrie par défaut d’une forme pédiatrique.ORPHELIA Pharma prévoit de publier les résultats de l’étude TEMOkids lors d’un congrès scientifique à venir et procèdera au dépôt du dossier d'autorisation de mise sur le marché auprès de l'Agence Européenne du Médicament en 2023.
- [Event :  BIO-EUROPE 2022 (Oct. 24-26, Leipzig, Germany)](https://www.orphelia-pharma.eu/news/event-bio-europe-2022-oct-24-26-leipzig-germany/): ORPHELIA Pharma will participate to BIO-EUROPE 2022 to be held from October 24-26, 2022 in Leipzig, Germany. This annual meeting aims to gather and connect biotechnology, pharmaceutical industry, and financial sector on pharmaceutical innovative projects.
- [Publication: dispensing temozolomide using KIMOZO or Temodal capsules mixed with food](https://www.orphelia-pharma.eu/news/publication-dispensing-temozolomide-using-kimozo-or-temodal-capsules-mixed-with-food/): ORPHELIA Pharma, the French pharmaceutical company that develops and commercializes orphan and pediatric medicines in the fields of oncology and neurology, announces the publication of the results of its study performed in collaboration with the Pharmacy Department of Gustave Roussy and evaluating the temozolomide dose actually delivered to children when using, on the one hand, KIMOZO oral suspension and, on the other hand, Temodal capsule content mixed with soft food or drinks.
- [SIOP 2022,  Barcelona, Spain : September 28-October 1, 2022](https://www.orphelia-pharma.eu/news/siop-2022-barcelona-spain/): ORPHELIA Pharma is proud to announce that it will attend 54th Annual SIOP Congress in Barcelona, Spain. M. Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma, will have the pleasure to present a poster entitled: “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures”.
- [Event :  European Paediatric Formulation Initiative – EuPFI  (Sept. 20-22, Rome, Italy)](https://www.orphelia-pharma.eu/news/event-european-paediatric-formulation-initiative-eupfi-sept-20-22-rome-italy/): ORPHELIA Pharma will participate to the 14th annual conference of The European Paediatric Formulation Initiative (EuPFI) to be held from September 20-22 in Rome, Italy. This annual meeting aims to gather industry, academia, clinical and regulatory professionals, to share expertise and interactive discussions on development of paediatric formulations.
- [Event: Organisation Inter Régionale Hémato-Onco pédiatrique (Sept. 19, Lille, France)](https://www.orphelia-pharma.eu/news/event-organisation-inter-regionale-hemato-onco-pediatrique-sept-19-lille-france/): ORPHELIA Pharma will participate to the first meeting "Organisation Inter Régionale Hémato-Onco pédiatrique Hauts-de-France and Grand-Est" to be held on Monday September 19, 2022 at the Faculty of Medicine of Lille, France.
- [1ère journée de l’Organisation Inter Régionale Hémato-Onco pédiatrique (19 septembre, Lille)](https://www.orphelia-pharma.eu/article-francais/1ere-journee-de-lorganisation-inter-regionale-hemato-onco-pediatrique-19-septembre-lille/): ORPHELIA Pharma participera à la première journée de l’Organisation Inter Régionale Hémato-Onco pédiatrique Hauts-de-France et Grand-Est qui se tiendra le lundi 19 septembre 2022 à la faculté de médecine de Lille (Pôle Recherche).
- [OCTALFA announces inclusion of the first patient in the French EAP of KIMOZO](https://www.orphelia-pharma.eu/news/octalfa-announces-the-inclusion-of-the-first-patient-in-the-french-early-access-program-granted-to-its-subsidiary-orphelia-pharma-for-the-use-of-kimozo-40-mg-ml-temozolomide-oral-suspension-in/): Lyon, June 22nd, 2022 – OCTALFA announces that its subsidiary ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of orphan medicines for the treatment of rare and pediatric diseases, has included the first patient in the KIMOZO® French Early Access Program for the treatment of relapsed or refractory neuroblastoma.
- [ORPHELIA Pharma et Gustave Roussy annoncent la publication dans la revue Pharmaceuticals des travaux de développement de la première suspension buvable de témozolomide pour les enfants](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-et-gustave-roussy-annoncent-la-publication-dans-la-revue-pharmaceuticals-des-travaux-de-developpement-de-la-premiere-suspension-buvable-de-temozolomide-pour-les-enfants/): Paris et Lyon, le 9 juin 2022 – ORPHELIA Pharma et Gustave Roussy annoncent conjointement aujourd’hui la publication dans la revue scientifique Pharmaceuticals de leurs travaux portant sur le développement d’une suspension buvable de témozolomide destinée au traitement de jeunes patients et d’adultes atteints d’un cancer et présentant des difficultés pour avaler les gélules de témozolomide (Annereau M. et al., Pharmaceuticals, 2022).
- [Publication in Pharmaceuticals of the first temozolomide oral suspension](https://www.orphelia-pharma.eu/news/orphelia-pharma-and-gustave-roussy-announce-the-publication-in-pharmaceuticals-of-the-scientific-work-related-to-the-development-of-the-first-temozolomide-oral-suspension-for-children/): Paris and Lyon, June 9th, 2022 – ORPHELIA Pharma and Gustave Roussy jointly announce today the publication in the scientific journal Pharmaceuticals of the work related to the development of the first temozolomide oral suspension for the treatment of young children and adults with cancer and presenting difficulties to swallow temozolomide capsules (Annereau M. et al., Pharmaceuticals, 2022).
- [Positive results of the bioequivalence study of Kimozo®](https://www.orphelia-pharma.eu/news/orphelia-reports-positive-results-of-the-bioequivalence-study-of-kimozo-oral-suspension-of-temozolomide/): Paris and Lyon, May 31st, 2022 – ORPHELIA Pharma, the French biopharmaceutical company focused on developing and commercializing orphan medicines in oncology and neurology, announces today the successful completion of the phase 1 study of KIMOZO®, novel ready-to-use oral suspension of temozolomide, aimed at determining bioequivalence between KIMOZO® suspension and temozolomide capsules.
- [ORPHELIA annonce les résultats positifs de l’étude de bioéquivalence de KIMOZO® (suspension orale de témozolomide)](https://www.orphelia-pharma.eu/article-francais/orphelia-annonce-les-resultats-positifs-de-letude-de-bioequivalence-de-kimozo-suspension-orale-de-temozolomide/): Paris et Lyon, le 31 mai 2022 – ORPHELIA Pharma, la société biopharmaceutique française dédiée au développement et à la commercialisation de médicaments pédiatriques dans les domaines de l'oncologie et de la neurologie, annonce aujourd’hui les résultats positifs de l'étude de bioéquivalence de KIMOZO®, nouvelle suspension orale de témozolomide prête à l'emploi. L'étude visait à déterminer la bioéquivalence entre la suspension KIMOZO® et les gélules de témozolomide.
- [ORPHELIA Pharma annonce l’obtention d’une aide de 1 million € de Bpifrance pour le développement d’un médicament destiné au traitement d’une forme rare d’épilepsie](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-lobtention-dune-aide-de-1-million-de-bpifrance-pour-le-developpement-dun-medicament-destine-au-traitement-dune-forme-rare-depilepsie/): Paris et Lyon, le 11 mai 2022 – ORPHELIA Pharma, la société biopharmaceutique française dédiée au développement et à la commercialisation de médicaments pédiatriques dans les domaines de l'oncologie et de la neurologie, annonce aujourd’hui avoir obtenu le soutien de Bpifrance à travers une avance récupérable d’un montant d’un million d’Euro. Cette aide a pour objectif d’accompagner le développement d’un médicament pour le traitement d’une encéphalopathie épileptique rare de l'enfant.
- [€ 1 million grant awarded to develop a therapy for a rare form of epilepsy](https://www.orphelia-pharma.eu/news/orphelia-pharma-has-been-awarded-a-e-1-million-grant-from-bpifrance-to-develop-a-therapy-for-a-rare-form-of-epilepsy/): Paris and Lyon, May 11, 2022 – ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of pediatric drugs in the fields of oncology and neurology, announces today that it has obtained a one million Euro grant from Bpifrance to develop a therapy to treat a rare epileptic encephalopathy in children.
- [International Childhood Cancer Day](https://www.orphelia-pharma.eu/news/international-childhood-cancer-day/): Today, February 15th 2022, is the International Childhood Cancer Day. ICCD is a global collaborative campaign to raise awareness about pediatric cancer and to express support for children and adolescents with cancer. At ORPHELIA Pharma, we are committed to improving cancer treatments for children. We are proud to announce that, during the annual symposium of Imagine for Margo - Children without Cancer (e-colloque FAST) held few days ago, we signed the charter aimed at accelerating the development of new drugs, facilitating the sharing of data for research and improving the long-term follow-up of patients. More information: https://lnkd.in/d9yApGWs ORPHELIA Pharma: https://lnkd.in/efSUdMP
- [Journée internationale des cancers de l’enfant](https://www.orphelia-pharma.eu/article-francais/journee-internationale-des-cancers-de-lenfant/): Aujourd’hui, 15 février 2022, est la journée internationale des cancers de l’enfant. Cette journée est une campagne mondiale de sensibilisation aux cancers pédiatriques et nous permet d’exprimer notre soutien aux enfants et adolescents atteints de cancer. Chez ORPHELIA Pharma, nous nous engageons à améliorer les traitements contre les cancers des enfants. Ainsi, nous sommes fiers d'annoncer que, lors du symposium annuel de l’association Imagine for Margo - Children without Cancer (e-colloque FAST) qui s'est tenu il y a quelques jours, nous avons signé la charte visant à accélérer le développement de nouveaux traitements, faciliter le partage et l’analyse des données pour une recherche dédiée et améliorer le suivi à long terme des patients. Plus d’informations : https://lnkd.in/d9yApGWs. ORPHELIA Pharma: https://lnkd.in/efSUdMP
- [World Cancer Day](https://www.orphelia-pharma.eu/news/world-cancer-day/): On the World Cancer Day, we stand in solidarity with patients and families affected by this disease. At ORPHELIA Pharma, we are committed to improving cancer treatments for children.  
- [ORPHELIA Pharma raises new money](https://www.orphelia-pharma.eu/news/orphelia-pharma-raises-new-money/): ORPHELIA Pharma raises new money to accelerate its commercial and clinical operations
- [ORPHELIA Pharma lève de nouveaux fonds](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-leve-de-nouveaux-fonds/): ORPHELIA Pharma lève de nouveaux fonds pour accélérer ses opérations commerciales et cliniques
- [ORPHELIA Pharma and K.S. KIM sign an agreement to supply Kigabeq®](https://www.orphelia-pharma.eu/news/orphelia-pharma-and-k-s-kim-sk-pharma-sign-an-agreement-to-supply-kigabeq/): Paris and Tel Aviv – November 16th 2021 – ORPHELIA Pharma and K.S. KIM INTERNATIONAL (SK-PHARMA) LTD announce today the execution of an exclusive distribution and marketing agreement for Kigabeq® (vigabatrin) in the territory of the Russian Federation.
- [SIOPEN annual meeting 2021: October 6-8th, 2021, in Barcelona](https://www.orphelia-pharma.eu/news/siopen-annual-meeting-2021-october-6-8th-2021-in-barcelona/): Jeremy Bastid and Hugues Bienayme will participate to the SIOPEN annual meeting to be held 6-8 October in Barcelona. Do not hesitate to contact us to meet.
- [<strong>ORPHELIA Pharma and Clinigen sign an agreement to supply Kigabeq®</strong>](https://www.orphelia-pharma.eu/news/orphelia-pharma-and-clinigen-sign-an-exclusive-agreement-to-supply-kigabeq/): Paris and Lyon, on July 21st, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs and Clinigen Group plc (AIM: CLIN, ‘Clinigen’), the global pharmaceutical Products and Services company, announce that they have signed an exclusive agreement to manage the supply and distribution of Kigabeq® into unlicensed markets.
- [European Patent covering Kimozo®](https://www.orphelia-pharma.eu/news/european-patent-covering-kimozo/): ORPHELIA Pharma and Gustave Roussy announce issuance of European Patent covering Kimozo®, the first oral liquid formulation of temozolomide
- [Délivrance d’un brevet européen couvrant Kimozo®](https://www.orphelia-pharma.eu/article-francais/delivrance-dun-brevet-europeen-couvrant-kimozo/): ORPHELIA Pharma et Gustave Roussy annoncent la délivrance d'un brevet européen couvrant Kimozo®, la première formulation liquide orale de témozolomide
- [Inclusion du premier patient dans l’essai clinique pédiatrique TEMOkids](https://www.orphelia-pharma.eu/article-francais/inclusion-du-premier-patient-dans-lessai-clinique-pediatrique-temokids/): ORPHELIA Pharma annonce l’inclusion du premier patient dans l’essai clinique pédiatrique TEMOkids
- [Inclusion of the first patient in the TEMOkids pediatric clinical trial](https://www.orphelia-pharma.eu/news/inclusion-of-the-first-patient-in-the-temokids-pediatric-clinical-trial-2/): ORPHELIA Pharma announces the inclusion of the first patient in the TEMOkids pediatric clinical trial
- [License agreement for the distribution of Kigabeq® in 14 european countries](https://www.orphelia-pharma.eu/news/license-agreement-for-the-distribution-of-kigabeq-in-14-european-countries/): ORPHELIA Pharma and Biocodex sign a license agreement for the distribution of Kigabeq® in 14 european countries
- [Accord de licence pour la distribution de Kigabeq® dans 14 pays européens](https://www.orphelia-pharma.eu/article-francais/accord-de-licence-pour-la-distribution-de-kigabeq-dans-14-pays-europeens/): ORPHELIA Pharma et Biocodex signent un accord de licence pour la distribution de Kigabeq® dans 14 pays européens
- [Opening of the operating pharmaceutical establishment](https://www.orphelia-pharma.eu/news/opening-of-the-operating-pharmaceutical-establishment-2/): ORPHELIA Pharma opens its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France
- [Ouverture de l’établissement pharmaceutique exploitant](https://www.orphelia-pharma.eu/article-francais/ouverture-de-letablissement-pharmaceutique-exploitant/): ORPHELIA Pharma annonce l’ouverture de son établissement pharmaceutique exploitant en France
- [Grant obtained from BPI to support activity development in EU and US](https://www.orphelia-pharma.eu/news/grant-obtained-from-the-region-ile-de-france-to-support-business-activity-development-in-europe-and-the-united-states/): ORPHELIA Pharma awarded a grant from the region Île-de-France to support business activity development in Europe and in the US
- [Obtention d’une subvention de la région Île-de-France pour soutenir le développement de son activité commerciale en Europe et aux États-Unis](https://www.orphelia-pharma.eu/article-francais/obtention-dune-subvention-de-la-region-ile-de-france-pour-soutenir-le-developpement-de-son-activite-commerciale-en-europe-et-aux-etats-unis/): ORPHELIA Pharma reçoit une subvention de la région Île-de-France pour soutenir le développement de son activité commerciale en Europe et aux États-Unis
- [Licensing agreement for the marketing of Kigabeq® in China](https://www.orphelia-pharma.eu/news/signature-of-a-licensing-agreement-for-the-development-and-marketing-of-kigabeq-in-china/): ORPHELIA Pharma and Ethypharm sign a licensing agreement for the development and marketing of Kigabeq® in China
- [Signature d’un contrat de licence pour le développement et la commercialisation de Kigabeq® en Chine](https://www.orphelia-pharma.eu/article-francais/signature-dun-contrat-de-licence-pour-le-developpement-et-la-commercialisation-de-kigabeq-en-chine/): ORPHELIA Pharma et Ethypharm signent un contrat de licence pour le développement et la commercialisation de Kigabeq® en Chine
- [ORPHELIA Pharma organise un Symposium lors du congrès SIOP 2020](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-organise-un-symposium-lors-du-congres-siop-2020-2/): Cher Docteur,
- [ORPHELIA Pharma to organize a Symposium at the SIOP congress 2020](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-to-organize-a-symposium-at-the-siop-congress-2020-2/): Dear Doctor,
- [ORPHELIA Pharma organise un Symposium lors du congrès SIOP 2020](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-organise-un-symposium-lors-du-congres-siop-2020/): Paris, le 12 octobre 2020 – ORPHELIA Pharma, société biopharmaceutique française dédiée au développement et à la commercialisation de médicaments pédiatriques et orphelins, annonce aujourd’hui l’organisation d’un symposium au congrès virtuel 2020 de la Société Internationale d’Oncologie Pédiatrique (SIOP). La séance se concentrera sur l’utilisation pédiatrique du témozolomide dans le traitement des tumeurs pédiatriques récidivantes ou réfractaires et mettra en valeur le développement de Kimozo, une suspension orale prête à l’emploi avec goût masqué de témozolomide adaptée aux enfants.
- [ORPHELIA Pharma to organize a Symposium at the SIOP congress 2020](https://www.orphelia-pharma.eu/news/orphelia-pharma-to-organize-a-symposium-at-the-siop-congress-2020/): Paris, October 12th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs announces today that it will organize a symposium at the Société Internationale d’Oncologie Pédiatrique (SIOP) congress 2020. The session will focus on the pediatric use of temozolomide in the treatment of relapsed or refractory pediatric tumors and will showcase the development of Kimozo a ready-to-use and taste-masked oral suspension of temozolomide adapted to children.
- [Inclusion d’un premier patient dans une étude clinique évaluant Kimozo](https://www.orphelia-pharma.eu/article-francais/inclusion-dun-premier-patient-dans-une-etude-clinique-evaluant-kimozo/): ORPHELIA Pharma annonce l’inclusion d’un premier patient dans une étude clinique évaluant Kimozo, sa formulation pédiatrique de témozolomide
- [Inclusion of the first patient in a clinical study evaluating Kimozo](https://www.orphelia-pharma.eu/news/inclusion-of-the-first-patient-in-a-clinical-study-evaluating-kimozo/): ORPHELIA announces the inclusion of the first patient in a clinical study evaluating Kimozo, its pediatric formulation of temozolomide
- [Meet us at BIO-Europe 2020, October 26-29](https://www.orphelia-pharma.eu/news/meet-us-at-bio-europe-2020-october-26-29/): Meet us at BIO-Europe 2020, October 26-29
- [ORPHELIA Pharma will attend and support the annual SIOP Congress 2020](https://www.orphelia-pharma.eu/news/orphelia-pharma-will-attend-and-support-the-annual-siop-congress-2020/): ORPHELIA Pharma will support and attend the upcoming SIOP Congress from October 14th to 17th (52nd Congress of the International Society of Paediatric Oncology), which will be virtual this year.
- [ORPHELIA Pharma annonce le recrutement de Laurent Martin au poste de Directeur des Affaires Pharmaceutiques](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-le-recrutement-de-laurent-martin-au-poste-de-directeur-des-affaires-pharmaceutiques/): Paris et Lyon, le 11 mars 2020 – ORPHELIA Pharma, société biopharmaceutique dont la mission est de développer et commercialiser des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, a le plaisir d’annoncer la nomination de Monsieur Laurent Martin au poste de Directeur des Affaires Pharmaceutiques, effective au 6 janvier 2020.
- [Appointment of L. Martin as Chief Pharmaceutical Affairs Officer](https://www.orphelia-pharma.eu/news/orphelia-announces-the-appointment-of-laurent-martin-as-chief-pharmaceutical-affairs-officer/): Paris and Lyon, March 11th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces the appointment of Laurent Martin as Chief Pharmaceutical Affairs Officer, effective January 6th, 2020.
- [Levée de fonds Orphelia Pharma (décembre 2019)](https://www.orphelia-pharma.eu/article-francais/levee-de-fonds-orphelia-pharma-decembre-2019/): Des Family Offices Français deviennent actionnaires et rejoignent le conseil d’administration
- [Orphelia Pharma fund raising](https://www.orphelia-pharma.eu/news/orphelia-pharma-fund-raising/): French family offices join as new shareholders and Board members
- [Launch of Kigabeq® in France](https://www.orphelia-pharma.eu/news/launch-of-kigabeq-in-france/): About infantile spasms
- [Lancement commercial de la spécialité Kigabeq® en France](https://www.orphelia-pharma.eu/article-francais/lancement-commercial-de-la-specialite-kigabeq-en-france/): Paris et Lyon, le 7 janvier 2020 – ORPHELIA Pharma, société biopharmaceutique qui développe et commercialise des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui la préparation du lancement de sa spécialité Kigabeq® après avoir conclu un accord avec le Comité Economique des Produits de Santé (CEPS).
- [ORPHELIA Pharma will be present at Toulouse for the thirtieth SFNP congress](https://www.orphelia-pharma.eu/news/orphelia-pharma-will-be-present-at-toulouse-for-the-thirtieth-sfnp-congress/): ORPHELIA Pharma will be present in Toulouse on January, 15th to 17th, for the SFNP Congress (Société Française de Neuropédiatrie). This event aims to bring together the French community of pediatric neurologists and share latest advances in paediatric neurology. Do not hesitate to contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu)
- [Obtention aux Etats-Unis d’une Désignation de Médicament Orphelin pour le témozolomide dans le traitement du neuroblastome](https://www.orphelia-pharma.eu/article-francais/obtention-aux-etats-unis-dune-designation-de-medicament-orphelin-pour-le-temozolomide-dans-le-traitement-du-neuroblastome/): ORPHELIA Pharma annonce l’obtention aux Etats-Unis d’une Désignation de Médicament Orphelin pour le témozolomide dans le traitement du neuroblastome
- [ODD for temozolomide in the treatment of neuroblastoma](https://www.orphelia-pharma.eu/news/orphan-drug-designation-granted-by-fda-for-temozolomide-in-the-treatment-of-neuroblastoma/): ORPHELIA Pharma announces Orphan Drug Designation granted by FDA for temozolomide in the treatment of neuroblastoma
- [Autorisation de Mise sur le Marché de la spécialité Ivozall](https://www.orphelia-pharma.eu/article-francais/autorisation-de-mise-sur-le-marche-de-la-specialite-ivozall/): ORPHELIA Pharma annonce avoir obtenu de la Commission Européenne l’Autorisation de Mise sur le Marché (AMM) de sa spécialité Ivozall®
- [Granting of the Marketing Authorisation for Ivozall](https://www.orphelia-pharma.eu/news/granting-of-the-marketing-authorisation-for-ivozall/): ORPHELIA Pharma announces the granting of the Marketing Authorisation for Ivozall® from the European Commission
- [ORPHELIA Pharma will be present at CPhI in Frankfurt](https://www.orphelia-pharma.eu/news/orphelia-pharma-will-be-present-at-cphi-in-frankfurt-2/): Caroline Lemarchand, Pharmaceutical Development Director of ORPHELIA Pharma will be present to the 2019 CPhI to be held in Frankfurt (Germany), November 5 - 7, 2019.
- [Accord de licence et de distribution pour Kigabeq au Royaume-Uni](https://www.orphelia-pharma.eu/article-francais/accord-de-licence-et-de-distribution-pour-kigabeq-au-royaume-uni/): ORPHELIA Pharma et Veriton Pharma annoncent la signature d’un accord de licence et de distribution pour Kigabeq® au Royaume-Uni
- [License and distribution agreement for Kigabeq in the United Kingdom](https://www.orphelia-pharma.eu/news/license-and-distribution-agreement-for-kigabeq-in-the-united-kingdom/): ORPHELIA Pharma and Veriton Pharma execute a license and distribution agreement for Kigabeq® in the United Kingdom
- [ORPHELIA at the Rare Diseases Meeting Days](https://www.orphelia-pharma.eu/news/orphelia-at-the-rare-diseases-meeting-days/): ORPHELIA Pharma will be
present in Paris on November, 5th and 6th, for the Rare Diseases Meeting Days.
This event is organized by the French Foundation for Rare Diseases every other
year, and allows to assist during two days to plenary conferences and
scientific round tables on current topics, poster presentations and meetings
with researchers, meeting with companies and stakeholders of the sector.   The organizing committee of the Foundation selected
ORPHELIA’s poster entitled “Multicentre retrospective study of temozolomide use
in paediatric refractory or relapsed neuroblastoma : Results from analysis of
data from French centres”,
to be presented during the Rare Diseases Meeting Days. Do not hesitate to
contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu).
- [ORPHELIA at the annual SIOP Congress](https://www.orphelia-pharma.eu/news/orphelia-at-the-annual-siop-congress/): ORPHELIA Pharma will be present in Lyon on October, 23th to 26th, for the annual SIOP Congress (the 51st Congress of the International Society for Paediatric Oncology). This event aims to bring together the oncology community and share latest advances in paediatric oncology. Do not hesitate to contact us in order to meet at this occasion (jeremy.bastid@orphelia-pharma.eu).
- [C.Lemarchand – Chief Pharmaceutical Development Officer](https://www.orphelia-pharma.eu/news/orphelia-announces-the-appointment-of-dr-caroline-lemarchand-as-chief-pharmaceutical-development-officer/): ORPHELIA announces the appointment of Dr. Caroline Lemarchand as Chief Pharmaceutical Development Officer
- [ORPHELIA Pharma annonce le recrutement du Dr. Caroline Lemarchand au poste de Directeur du Développement Pharmaceutique](https://www.orphelia-pharma.eu/article-francais/caroline-lemarchand-au-poste-de-directeur-du-developpement-pharmaceutique/): Paris et Lyon, le 15 octobre 2019 – ORPHELIA Pharma, société biopharmaceutique dont
la mission est de développer et commercialiser des médicaments pédiatriques
dans les domaines de la neurologie et de l’oncologie, a le plaisir d’annoncer la
nomination de Madame Caroline Lemarchand au poste de Directeur du Développement Pharmaceutique, effective au 16 septembre 2019. Pharmacien
et scientifique de formation, Caroline Lemarchand possède plus de 20 années
d’expérience dans le développement de médicaments, particulièrement en
oncologie, au sein de l’industrie pharmaceutique et de sociétés de
biotechnologie. Caroline a notamment dirigé le développement préclinique et
pharmaceutique chez Onxeo (Bioalliance Pharma), avant de prendre la direction du
développement pharmaceutique chez Stallergenes Greer. Caroline
Lemarchand a conduit le développement, l’enregistrement et la commercialisation
en Europe et aux Etats-Unis d’une dizaine de médicaments (formulations
innovantes, nouvelles entités chimiques, biologiques). Elle est l’auteur de plusieurs
publications scientifiques et brevets. « Nous
sommes particulièrement heureux d’accueillir Caroline au sein de notre entreprise.»
commente Hugues Bienaymé, Directeur Général d’ORPHELIA Pharma. « Sa grande
expérience du développement est un atout précieux pour une entreprise comme
ORPHELIA Pharma, qui se focalise sur des maladies graves mettant en jeu la vie des
enfants. Caroline aura un rôle clé dans la gestion de nos partenariats afin
de constituer un portefeuille de médicaments pédiatriques.» « Je
suis ravie de rejoindre ORPHELIA Pharma à ce point d'inflexion important pour
la société avec déjà 2 produits approuvés et plusieurs projets collaboratifs en
cours » indique Caroline Lemarchand. « La
société est dans une position privilégiée pour constituer un portefeuille de
médicaments destinés aux enfants, à la fois en neurologie et en oncologie. De
nombreuses collaborations sont déjà en place et je suis donc impatiente de
diriger ces partenariats et en faire émerger de nouveaux. » A propos
d’ORPHELIA Pharma ORPHELIA Pharma est une société biopharmaceutique basée à Paris et à
Lyon qui développe et commercialise des médicaments pour le traitement de
pathologies rares et graves de l’enfant. Sa mission est de mettre à disposition
des patients des produits hospitaliers essentiels dans les domaines de l’épilepsie
et de l’oncologie avec une formulation adaptée à la pédiatrie. Son premier
produit, Kigabeq®, a obtenu une AMM européenne en octobre 2018 et est en phase
de lancement en Europe. Le deuxième produit d’ORPHELIA Pharma, Ivozall®, a reçu
en Septembre 2019 une opinion positive du Comité des Médicaments à usage humain
(CHMP) pour l’octroi de l’autorisation de mise sur le marché par la Commission
Européenne. ORPHELIA Pharma conduit des projets de recherche innovants à
travers des collaborations académiques et industrielles. Ses investisseurs
principaux sont initiative OCTALFA et Pierre Fabre Médicament.
- [Opinion positive du CHMP pour Ivozall®](https://www.orphelia-pharma.eu/article-francais/opinion-positive-du-chmp-pour-ivozall/): ORPHELIA Pharma annonce avoir reçu une opinion positive du CHMP pour Ivozall® (clofarabine 1mg/ml), un médicament indiqué dans la Leucémie Aiguë Lymphoblastique réfractaire ou en rechute chez l’enfant
- [CHMP has adopted a positive opinion for Ivozall®](https://www.orphelia-pharma.eu/news/chmp-has-adopted-a-positive-opinion-for-ivozall/): ORPHELIA Pharma announces that the CHMP has adopted a positive opinion for Ivozall® (clofarabine 1 mg/ml), an essential medicine intended for the treatment of relapsed or refractory Acute Lymphoblastic Leukaemia in children
- [ORPHELIA at the XIIth National Oncology Days](https://www.orphelia-pharma.eu/news/orphelia-recrute-un-pharmacien-responsable-2-2-2/): ORPHELIA Pharma will be present in Avignon on October, 9th to 11th, for the XIIth National Oncology Days of the French Society of Pharmacy Oncology. This event aims to bring together the pharmaceutical oncology community and inform them of advances in oncology treatments. Do not hesitate to contact us at this occasion (jeremy.bastid@orphelia-pharma.eu) and visit our exhibition booth at the “Palais des papes” in Avignon (booth 47 - "Paneterie" room).
- [Désignation orpheline pour le témozolomide dans le traitement du neuroblastome](https://www.orphelia-pharma.eu/article-francais/designation-orpheline/): ORPHELIA Pharma annonce l’obtention en Europe d’une Désignation de Médicament Orphelin pour le témozolomide dans le traitement du neuroblastome
- [ODD-EU for temozolomide in the treatment of neuroblastoma](https://www.orphelia-pharma.eu/news/orphan-drug-designation/): ORPHELIA Pharma announces Orphan Drug Designation granted by EMA for temozolomide in the treatment of neuroblastoma
- [ORPHELIA at the 22nd French Epilepsy Days](https://www.orphelia-pharma.eu/news/orphelia-recrute-un-pharmacien-responsable-2-2/): ORPHELIA Pharma will be present in Paris on October, 7th to 10th, for the 22nd French Epilepsy Days. This congress is schedule by the “Ligue Française contre l'Épilepsie “ (LFCE), French subsidiary of the International Ligue Against Epilepsy (ILAE). Do not hesitate to contact us in order to meet at this occasion (hugues.bienayme@orphelia-pharma.eu).
- [ORPHELIA recrute un Responsable Affaires Règlementaires](https://www.orphelia-pharma.eu/news/orphelia-recrute-un-pharmacien-responsable-2/): Recrutement d'un Pharmacien(ne) en charge des Affaires Règlementaires :
- [Commercialisation de Kigabeq® en Allemagne](https://www.orphelia-pharma.eu/article-francais/commercialisation-de-kigabeq-en-allemagne/): ORPHELIA Pharma et DESITIN GmbH annoncent le lancement de Kigabeq® en Allemagne
- [Kigabeq® launch in Germany](https://www.orphelia-pharma.eu/news/kigabeq-launch-in-germany/): ORPHELIA Pharma and DESITIN GmbH announce the launch of Kigabeq® in Germany
- [Gustave Roussy Cancer Centre and Orphelia Pharma](https://www.orphelia-pharma.eu/news/collaboration-between-gustave-roussy-cancer-centre-and-orphelia-pharma/): ORPHELIA Pharma executes a collaboration and license agreement with Gustave Roussy cancer centre for the development of the first pediatric formulation of temozolomide
- [Accord de collaboration entre ORPHELIA Pharma et Gustave Roussy](https://www.orphelia-pharma.eu/article-francais/collaboration-orphelia-pharma-et-gustave-roussy/): ORPHELIA Pharma et Gustave Roussy signent un accord de collaboration pour le développement de la première formulation pédiatrique de témozolomide
- [ORPHELIA recrute un Pharmacien Responsable](https://www.orphelia-pharma.eu/news/orphelia-recrute-un-pharmacien-responsable/): Recrutement d'un Pharmacien Responsable et Directeur Assurance Qualité (Qualified Person & Quality Assurance Director) :
- [ORPHELIA at the 2019 ASCO meeting in Chicago](https://www.orphelia-pharma.eu/news/orphelia-at-the-2019-asco-meeting-in-chicago/): The Annual Congress of the American Society of Clinical Oncology will take place May 31 - June 4, 2019 at McCormick Place, Chicago, IL.
- [ORPHELIA at the 2018 SIOP meeting in Kyoto (Japan)](https://www.orphelia-pharma.eu/news/orphelia-at-the-2018-siop-meeting-in-kyoto-japan/): The 50th Annual Congress of the International Society of Paediatric Oncology will take place November 16-19, 2018 in Kyoto, Japan.
- [ORPHELIA at Bioeurope in Copenhagen, Denmark](https://www.orphelia-pharma.eu/non-classe/orphelia-at-bioeurope-in-copenhagen-denmark/): ORPHELIA Pharma will be present in Copenhagen on November 2018, 5th to 7th, for the annual BIO-Europe Congress. This event is the Europe’s largest partnering conference serving the global biotechnology industry. Do not hesitate to contact us in order to meet at this occasion (contact : gilles.alberici@orphelia-pharma.eu).
- [ORPHELIA Pharma annonce l’obtention d’une aide Bpifrance](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-lobtention-dune-aide-bpifrance/): Paris et Lyon, le 22 octobre 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est de développer et commercialiser des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui avoir obtenu le soutien de Bpifrance à travers un prêt FEDER (Fonds Européen de Développement Régional) innovation à taux zéro d’un montant d’un million d’Euros cofinancé par l’Union Européenne et la région Auvergne Rhône-Alpes. Ce prêt a pour objectif d’accompagner le développement et l’industrialisation de la première formulation pédiatrique d’un médicament essentiel. Le développement porte sur le traitement de rechute d’un cancer rare de l’enfant. « Nous nous réjouissons du soutien de Bpifrance qui nous permet d’accélérer le développement de ce médicament dans le traitement cette indication pédiatrique dont le besoin médical est considérable », commente Jérémy Bastid, Directeur du Développement d’ORPHELIA Pharma. « Ce médicament, qui possède une formulation spécialement adaptée aux jeunes enfants, est développé en étroite collaboration avec les cliniciens depuis 2017. Cette aide participera au coût de développement du produit jusqu’à sa mise à disposition des patients d’ici 2 à 3 ans », ajoute Hugues Bienaymé, Directeur Général.
- [ORPHELIA Pharma has been awarded a €1m loan from Bpifrance](https://www.orphelia-pharma.eu/news/orphelia-pharma-has-been-awarded-a-e1m-loan-from-bpifrance/): Paris and Lyon, October 22, 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 obtained the support of Bpifrance through a ERDF (European Regional Development Fund) innovation loan of one million Euros co-financed by the European Union and the Auvergne Rhône-Alpes region. This grant aims to support the development and industrialization of the first pediatric formulation of an essential drug as a salvage therapy of a rare pediatric cancer. "We are delighted to receive the support from Bpifrance which allows us to accelerate the development of our product in this pediatric indication whose unmet medical need is huge", comments Jérémy Bastid, Chief Development Officer of ORPHELIA Pharma. "This product has a formulation specially adapted for young children and has been developed in close collaboration with clinicians since 2017. This grant will contribute to the development cost of the product until it becomes available to patients in 2 to 3 years", adds Hugues Bienaymé, General Manager.
- [Autorisation de mise sur le marché pour Kigabeq®](https://www.orphelia-pharma.eu/article-francais/autorisation-de-mise-sur-le-marche-pour-kigabeq/): Paris et Lyon, le 1er octobre 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est de développer et commercialiser des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui avoir reçu de la Commission Européenne la décision d’exécution accordant l’autorisation de mise sur le marché Européen de sa spécialité Kigabeq® .
- [Granting of the marketing authorization for Kigabeq®](https://www.orphelia-pharma.eu/news/granting-of-the-marketing-authorization-for-kigabeq/): Paris and Lyon, October 1st, 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 European Commission has granted Kigabeq® a European marketing authorization.
- [ORPHELIA Pharma will be present at CPhI in Madrid](https://www.orphelia-pharma.eu/news/orphelia-pharma-will-be-present-at-cphi-in-madrid/): Hugues Bienaymé, General Manager and Chief Scientific Officer of ORPHELIA Pharma and Mathieu Schmitt, Pharmaceutical Development Director, will be present to the 2018 CPhI to be held in Madrid, Spain, October 9-11, 2018.
Please meet us there (contact: mathieu.schmitt@orphelia-pharma.eu).
- [Opinion positive du CHMP pour Kigabeq®, la première formulation pédiatrique de vigabatrine](https://www.orphelia-pharma.eu/article-francais/opinion-positive-du-chmp-pour-kigabeq-la-premiere-formulation-pediatrique-de-vigabatrine/): Paris et Lyon, le 28 aout 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est de développer et commercialiser des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui avoir reçu une opinion positive du Comité des Médicaments à usage humain (CHMP) de l’Agence Européenne du Médicament (EMA) pour l’octroi de l’autorisation de mise sur le marché de sa spécialité Kigabeq® par la Commission Européenne.
- [CHMP has adopted a positive opinion for Kigabeq®](https://www.orphelia-pharma.eu/news/chmp-has-adopted-a-positive-opinion-for-kigabeq-the-first-pediatric-formulation-of-vigabatrin/): 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).
- [ORPHELIA Pharma annonce la nomination de Pierre Attali  en tant que membre de son Conseil d’Administration](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-la-nomination-de-pierre-attali-en-tant-que-membre-de-son-conseil-dadministration/): Paris et Lyon, le 25 juin 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est de développer et commercialiser des médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, a le plaisir d’annoncer que Monsieur Pierre Attali rejoint son Conseil d’Administration.
- [Pierre Attali joins ORPHELIA Pharma Board Of Directors](https://www.orphelia-pharma.eu/news/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.
- [ORPHELIA at the 2018 BIO Convention in Boston](https://www.orphelia-pharma.eu/news/orphelia-at-the-2018-bio-convention-in-boston/): Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the 2018 BIO International Convention, to be held in Boston, Massachusetts, June 4-7, 2018.
- [ORPHELIA at the 2018 ASCO annual meeting in Chicago](https://www.orphelia-pharma.eu/news/orphelia-at-the-2018-asco-annual-meeting-in-chicago/): Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the 2018 ASCO annual meeting to be held in Chicago, Illinois, June 1-5, 2018.
- [ORPHELIA at the 2018 ANR meeting in San Francisco](https://www.orphelia-pharma.eu/news/orphelia-at-the-2018-anr-meeting-in-san-francisco/): Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will attend the Advances in Neuroblastoma Research (ANR) 2018 conference, “Building Bridges for Neuroblastoma Research” in San Francisco, California from May 9-12, 2018.
- [ORPHELIA Pharma annonce le dépôt du dossier d’enregistrement européen de Kigabeq®, la première formulation pédiatrique de vigabatrine](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-annonce-depot-dossier-denregistrement-europeen-de-kigabeq-premiere-formulation-pediatrique-de-vigabatrine/): Paris et Lyon, le 27 février 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est le développement et la commercialisation de médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui avoir déposé en 2017 un dossier de demande d’autorisation de mise sur le marché, en procédure centralisée pour sa spécialité Kigabeq®.
- [ORPHELIA announces the filing of the Marketing Authorization for Kigabeq®](https://www.orphelia-pharma.eu/news/orphelia-pharma-announces-the-filing-of-a-european-marketing-authorization-application-for-kigabeq-the-first-pediatric-formulation-of-vigabatrin/): 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.
- [On parle de nous!](https://www.orphelia-pharma.eu/news/on-parle-de-nous/): Orphelia Pharma accélère grâce à une levée de fonds
- [ORPHELIA PHARMA strengthens its capital base](https://www.orphelia-pharma.eu/news/orphelia-pharma-strengthens-its-capital-base-and-accelerates-its-development/): 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.
- [ORPHELIA PHARMA renforce ses fonds propres et accélère son développement](https://www.orphelia-pharma.eu/article-francais/orphelia-pharma-renforce-ses-fonds-propres-et-accelere-son-developpement/): Paris, le 30 janvier 2018 – ORPHELIA Pharma, société biopharmaceutique dont la mission est le développement et la commercialisation de médicaments pédiatriques dans les domaines de la neurologie et de l’oncologie, annonce aujourd’hui qu’elle a renforcé ses fonds propres en décembre 2017. Cette levée de fonds, souscrite intégralement par initiative OCTALFA, permet d’accélérer les projets de la société avec l’objectif ambitieux d’obtenir trois enregistrements de médicaments pédiatriques en Europe avant la fin de l’année 2020.
- [ORPHELIA at the 2017 SIOP meeting in Washington DC](https://www.orphelia-pharma.eu/news/orphelia-pharma-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/
- [ORPHELIA Pharma at AES 2017 in Washington DC](https://www.orphelia-pharma.eu/news/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.
- [ORPHELIA Pharma et BIO-Europe 2017](https://www.orphelia-pharma.eu/news/orphelia-pharma-et-bio-europe-2017/): ORPHELIA Pharma will be present in Berlin on November, 6th to 8th, for the annual BIO-Europe Congress. This event is the Europe’s largest partnering conference serving the global biotechnology industry. Do not hesitate to contact us in order to meet at this occasion.
- [ORPHELIA Pharma will be present at CPhI in Frankfurt](https://www.orphelia-pharma.eu/news/orphelia-pharma-will-be-present-at-cphi-in-frankfurt/): ORPHELIA Pharma will be present in Frankfurt on October 24th to 25th, 2017, in the number one Pharma event that brings together most international pharmaceuticals companies. Do not hesitate to contact us to meet at this occasion.
- [ORPHELIA Pharma at CPhI, Barcelona – Spain](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-cphi-barcelona-spain/): ORPHELIA Pharma will be present in Barcelona on October 4th to 6th, 2016, in the number one Pharma event that brings together most international pharmaceuticals companies. Do not hesitate to contact us to meet at this occasion.
- [ORPHELIA Pharma at BIO, San Francisco, USA](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-bio-san-francisco-usa/): ORPHELIA Pharma will be present in San Francisco on June, 6 to 9, for the BIO International Convention. The BIO International Convention attracts over 15,000 biotechnology and pharma leaders who come together for one week of intensive networking to discover new opportunities and promising partnerships. Do not hesitate to contact us in order to meet at this occasion.
- [ORPHELIA Pharma at Bio-Europe, Stockholm, Sweden](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-bio-europe-stockholm-sweden/): ORPHELIA Pharma will be present in Stockholm on April, 4 to 6, for the annual BIO-Europe Congress. This event is the Europe’s largest partnering conference serving the global biotechnology industry. Do not hesitate to contact us in order to meet at this occasion.
- [ORPHELIA Pharma joins MEDICEN Paris Région](https://www.orphelia-pharma.eu/news/orphelia-pharma-joins-medicen-paris-region/): ORPHELIA Pharma joins MEDICEN Paris Région as an active member. The competitive cluster MEDICEN Paris Région, which is dedicated to new technologies and innovative therapies, and of SMEs, contributes since its creation in 2006 to boost growth and influence of the Ile-de-France health sector.
- [ORPHELIA Pharma strengthens its development operations](https://www.orphelia-pharma.eu/news/orphelia-pharma-strengthens-its-development-operations/): ORPHELIA Pharma, a French company specialized in the development and commercialization of pediatric medicines, today announced that initiative Octalfa joined the company as a minority shareholder, director and operating partner.
- [TargeOn SAS to become ORPHELIA Pharma SA](https://www.orphelia-pharma.eu/news/targeon-sas-to-become-orphelia-pharma-sa/): TargeOn SAS, a French company specialized in the development and commercialization of pediatric medicines, today announced that the company will be renamed ORPHELIA Pharma SA, effective October 9th, 2015.
- [ORPHELIA Pharma at Bio-Europe, Munich – Germany](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-bio-europe-munich-germany/): ORPHELIA Pharma will be present in Munich on November, 2 to 4 for the 21st annual BIO-Europe Congress. This event is the Europe's largest partnering conference serving the global biotechnology industry. Do not hesitate to contact us to meet at this occasion.
- [ORPHELIA Pharma at RARE 2015, Montpellier – France](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-rare-2015-montpellier-france/): ORPHELIA Pharma will be present in Montpellier on November 26th and 27th for the RARE congress. This event brings together manufacturers, researchers, clinicians, patient organizations, regulatory authorities, and investors involved in the field of rare diseases. Do not hesitate to contact us to meet at this occasion.
- [ORPHELIA Pharma at CPhI, Madrid – Spain](https://www.orphelia-pharma.eu/news/orphelia-pharma-at-cphi-madrid-spain/): ORPHELIA Pharma will be present in Madrid on October 13th and 14th, 2015, in the number one Pharma event that brings together most international pharmaceuticals companies. Do not hesitate to contact us to meet at this occasion.

## Pages
- [Pipeline](https://www.orphelia-pharma.eu/pipeline/): Pipeline
- [Oncology](https://www.orphelia-pharma.eu/oncology/): Kizfizo 40 mg/ml is a ready-to-use and taste-masked oral suspension of temozolomide.
- [Neurology](https://www.orphelia-pharma.eu/neurology/): Kigabeq® is a pediatric form of vigabatrin (soluble tablets).
- [About us](https://www.orphelia-pharma.eu/about-us/): ORPHELIA Pharma is a French pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric orphan diseases.
- [Contact](https://www.orphelia-pharma.eu/contact/): To report any adverse reaction (side effect) or suspicion of adverse reaction associated with an ORPHELIA Pharma medicine, please contact us at: pv@orphelia-pharma.eu
- [News](https://www.orphelia-pharma.eu/news-and-events/): 30 octobre 2023
- [Partners](https://www.orphelia-pharma.eu/partners/): Since 2018, Orphelia Pharma has been working in close collaboration with Gustave Roussy, one of the largest cancer centers in Europe, on developing a liquid oral formulation of temozolomide.
- [Homepage](https://www.orphelia-pharma.eu/): Four key pillars :