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.
ANR meetings gather investigators from around the world studying neuroblastoma biology, diagnosis, prognosis, and therapy.
If you would like to arrange a meeting with Jeremy, please contact him at: jeremy.bastid@orphelia-pharma.eu
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/).
We are pleased to announce that Jeremy Bastid, Chief Development Officer, and Hugues Bienaymé, General Manager and Chief Scientific Officer at ORPHELIA Pharma, will attend SIOP Europe 2023. We will be present at booth #10 located near the entrance doors to Auditorium 1. Our booth will showcase the scientific data of our paediatric temozolomide oral suspension (KIMOZO).
If you would like to meet Jeremy or Hugues, please contact them at: jeremy.bastid@orphelia-pharma.eu or hugues.bienayme@orphelia-pharma.eu
KIMOZO 40 mg/ml is a ready-to-use and taste-masked oral suspension of temozolomide. KIMOZO is being developed by ORPHELIA Pharma for the treatment of relapsed or refractory neuroblastoma in pediatric patients aged 12 months and above. KIMOZO is not an approved medicinal product and is made available in certain European countries only via a named patient access program by ORPHELIA Pharma or Tanner Pharma (www.tannerpharma.com).
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.
Temozolomide is an anti-cancer drug approved as capsules or IV formulation in Europe for certain brain tumors. It is also used in treatment protocols for other rare cancer indications that mainly affect very young children, including refractory or relapsed neuroblastoma. In young children, an oral suspension of temozolomide is age-appropriate to ensure correct medication administration.
Under the terms of the agreement, Tanner Pharma will provide access to KIMOZO® via a named patient program in European countries, in particular these countries where KIMOZO® has already been used as investigational medication in the context of the now fully enrolled clinical trial TEMOkids sponsored by ORPHELIA Pharma. A named patient program is a mechanism through which physicians can legally and ethically prescribe investigational or approved drugs for patients prior to their commercial availability. The access to unapproved drugs is strictly regulated and adherence to national regulations is mandatory.
Tanner Pharma will manage all elements of the supply including healthcare practitioner enquiry management, national regulatory oversight, liaison with national authorities, and logistics. The agreement currently covers all European territories but France.
Hugues Bienaymé, General Manager of ORPHELIA Pharma, said:
“KIMOZO® is currently available in France through an early access program, and ORPHELIA has already received requests from physicians from other countries. To meet these demands, ORPHELIA Pharma will use the services of Tanner Pharma, a world-leading pharmaceutical services provider with extensive experience in enabling access to innovative therapies when no other treatment options exist. With this agreement, our ambition is to make KIMOZO® available to children in need of a ready-to-use drinkable suspension of temozolomide, anywhere in Europe and with no delay, in strict adherence with local regulations.”
Rob Keel, Executive Vice President at Tanner Pharma, said:
“We are pleased to be partnering with ORPHELIA Pharma on this impactful program to help children with neuroblastoma and other malignancies. ORPHELIA Pharma has done extensive research to develop a product to address a critical unmet need for pediatric patients. We look forward to supporting physicians and enabling access to this innovative treatment.”
Healthcare professionals can obtain details about the products by contacting: kimozo@tannerpharma.com
About KIMOZO® 40 mg/ml
KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use drinkable formulation of temozolomide developed in collaboration between the pharmacists and clinicians of Gustave Roussy hospital and the development team of ORPHELIA Pharma. KIMOZO® is being developed for the treatment of refractory and relapsed neuroblastoma, a childhood cancer of dismal prognosis.
KIMOZO® has been granted Early Access Authorization (Autorisation d’Accès Précoce) for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with a specific DNA inhibitor topoisomerase I (irinotecan or topotecan) in patients aged 1 to 6 years and patients over 6 years of age unable to swallow temozolomide in capsule form, by the French authorities in March 2022.
The formulation of KIMOZO® has been covered by a patent application in Europe and in the US.
About ORPHELIA Pharma
ORPHELIA Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.
About Tanner Pharma
Tanner Pharma Group is a specialist pharmaceutical services provider based in Charlotte, USA with offices in Europe and Latin America. Over the past 20 years, Tanner has developed a portfolio of service offerings focused on improving global access to medicines. Through its TannerMAP division, Tanner Pharma supports manufacturers by providing ethical, controlled and compliant access to their innovative medicines in countries where they are not commercially accessible. For more information, visit www.tannerpharma.com.
Media Contact details
ORPHELIA Pharma / Magali Rosbif – orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18
Tanner Pharma Group / Lindsey Stevens – lstevens@tannerpharma.com / Tel: +1 704 552 8408
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.
« Nous concluons le recrutement de l’étude clinique TEMOkids avec l’implication et la motivation des médecins investigateurs dans cet essai. Grâce à l’appui d’un réseau structuré d’oncologues pédiatres au niveau européen, nous avons recruté 43 enfants dans 11 centres et 5 pays : France, Espagne, Royaume-Uni, Pays-Bas et Allemagne », déclare Caroline Lemarchand, Directrice du Développement Pharmaceutique et coordinatrice de l’étude TEMOkids.
« L’inclusion du dernier patient dans TEMOkids marque une étape-clé du développement clinique de KIMOZO®. Cette nouvelle formulation buvable de témozolomide comble un besoin médical majeur pour la prise en charge des cancers du jeune enfant », conclut Jeremy Bastid, Directeur du Développement d’ORPHELIA Pharma. « Notre souhait est désormais de mettre KIMOZO® à disposition de tous les patients le plus rapidement possible. Toute l’équipe d’ORPHELIA Pharma est mobilisée pour permettre le dépôt du dossier européen d’enregistrement en 2023 et pour mettre en place dans les meilleurs délais des programmes d’accès spécial, en liaison étroite avec les autorités réglementaires nationales en Europe et avec leur approbation », ajoute-t-il.
À propos de l’étude TEMOkids
L’essai clinique intitulé « TEMOkids Study : A Phase I Pediatric Study for KIMOZO®, Oral Suspension of temozolomide » (NCT04610736) est une étude internationale de phase I, ouverte, non randomisée chez 40 patients pédiatriques âgés d’un an ou plus. L’objectif principal de l’étude est d’évaluer les paramètres pharmacocinétiques de KIMOZO®/Ped-TMZ dans la population de l’étude. Les objectifs secondaires sont d’évaluer la tolérance et la réponse au traitement, ainsi que l’acceptabilité chez les enfants.
Onze centres cliniques sont activement impliqués dans l’étude TEMOkids : Gustave Roussy, Villejuif, France (centre coordinateur) ; Institut Curie, Paris, France ; CHU Timone Enfants, Marseille, France ; Institut d’Hématologie et d’Oncologie Pédiatrique, Lyon, France ; Centre Oscar Lambret, Lille, France ; Princess Maxima Center for Pediatric Oncology, Utrecht, Pays-Bas ; Hospital Universitari Vall d’Hebron, Barcelone, Espagne ; Hospital La Fe, Valence, Espagne ; University Pediatric Hospital Niño Jesús, Madrid, Espagne ; Southampton General Hospital, Southampton, Royaume-Uni ; Great Ormond Street Hospital (GOSH) for Children NHS Trust, Londres, Royaume-Uni; hôpital de la Charité, Berlin, Allemagne.
Pour plus d’informations, veuillez visiter www.temokids.eu.
À propos de KIMOZO®/Ped-TMZ suspension buvable, 40 mg/ml
KIMOZO® 40 mg/ml est une formulation de témozolomide buvable, au goût masqué et prête à l’emploi, développée conjointement par les pharmaciens et cliniciens de l’hôpital Gustave Roussy et l’équipe de développement d’ORPHELIA Pharma. KIMOZO® est en cours de développement pour le traitement du neuroblastome réfractaire et récidivant, un cancer infantile au pronostic sombre.
KIMOZO® a obtenu une Autorisation d’Accès Précoce des autorités françaises en mars 2022.
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 des maladies pédiatriques et orphelines. La mission d’ORPHELIA Pharma est de fournir aux patients des produits essentiels dans les domaines des maladies rares, sous des formulations adaptées à la population pédiatrique. Avec deux médicaments approuvés dans l’Union Européenne et un produit en phase avancée de développement clinique, ORPHELIA Pharma a récemment mis en place des accords régionaux dans l’Union Européenne, aux USA et en Chine et mène des projets de recherche grâce à des partenariats académiques et industriels.
Plus d’information sur www.orphelia-pharma.eu.
Contacts : Magali Rosbif / orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18
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.
“We have completed the enrollment of the TEMOkids clinical study thanks to the involvement and motivation of the investigating physicians, which underlines the medical need. With the support of a pediatric oncology network at the European level, we enrolled 43 children in 11 centers across 5 countries: France, Spain, the United Kingdom, the Netherlands and Germany“, says Caroline Lemarchand, Director of Pharmaceutical Development and coordinator of the TEMOkids study.
“The inclusion of the last patient in TEMOkids is a key milestone of the clinical development of KIMOZO®. This new drinkable formulation of temozolomide addresses major medical needs for the management of cancers affecting young children,” concludes Jeremy Bastid, Director of Development at ORPHELIA Pharma. “Our goal is to make the product available to all patients as soon as possible. The ORPHELIA Pharma team is mobilized to enable the filing of the European registration dossier in 2023 and to fill the needs of young patients through special access programs, in close liaison with and with the approval of the national regulatory authorities in Europe,” he adds.
About the TEMOkids study
The clinical trial “TEMOkids Study: A Phase I Pediatric Study for KIMOZO®, Oral Suspension of temozolomide®” (NCT04610736) is an international, open-label, non-randomized Phase I study in 40 pediatric patients aged one year or older. The primary objective of the study is to evaluate the pharmacokinetics of KIMOZO®/Ped-TMZ in the study population. Secondary objectives are to assess tolerability and response to treatment, as well as acceptability in children. Eleven clinical centers are actively involved in the TEMOkids study: Gustave Roussy, Villejuif, France (coordinating center); Institut Curie, Paris, France; CHU Timone Enfants, Marseille, France; Institute of Hematology and Pediatric Oncology, Lyon, France; Centre Oscar Lambret, Lille, France; Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands; Hospital Universitari Vall d’Hebron, Barcelona, Spain; Hospital La Fe, Valencia, Spain; University Pediatric Hospital Niño Jesús, Madrid, Spain; Southampton General Hospital, Southampton, United Kingdom; Great Ormond Street Hospital (GOSH) for Children NHS Trust, London, United Kingdom; Charité University Medicine Hospital, Berlin, Germany.
For more information, please visit www.temokids.eu.
About KIMOZO®/Ped-TMZ oral suspension, 40 mg/ml
KIMOZO® 40 mg/ml is a ready-to-use, taste-masked oral liquid formulation of temozolomide jointly developed by the ORPHELIA Pharma development team and the pharmacists and clinicians of Gustave Roussy for the treatment of relapsed or refractory neuroblastoma, a childhood cancer with a poor prognosis.
The product obtained an Early Access Authorization from the French authorities in March 2022.
About ORPHELIA Pharma
ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of rare diseases, with formulations adapted to the pediatric population. With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.
More information on www.orphelia-pharma.eu.
Contacts: Magali Rosbif / orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18
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.
We are glad to announce that Gilles Alberici, Executive Chairman and Hugues Bienaymé, General Manager and Chief Scientific Officer at ORPHELIA Pharma, will attend this event. If you would like to meet Gilles and Hugues, please contact them : contact@orphelia-pharma.eu.
Sponsored by : La Région Auvergne – Rhône – Alpes
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.
The poster #128 entitled “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures” (Lemarchand et al.), is displayed during the poster session of the SIOP 2022 Annual Congress held in Barcelona (Spain). The poster can also be accessed by using this link.
About SIOP
SIOP (International Society of Pediatric Oncology) is the only multidisciplinary and international learned society entirely dedicated to childhood and adolescent cancer. The society has more than 2,600 members worldwide, including doctors, nurses, other health professionals, scientists and researchers. Members are dedicated to increasing knowledge about all aspects of childhood cancer (https://siop-online.org/). The annual congress is held in Barcelona, Spain, from 28th September to 1st October, 2022 (https://siop-congress.org/).
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”.
The SIOP 2022 Annual Meetings will bring together more than 3,000 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. The SIOP 2020 Annual Meeting will take place in Barcelona, Spain, from September 28-October 1, 2022 (https://siop-congress.org/).
Hugues Bienaymé, General Manager, and Jeremy Bastid, Chief Development Officer at OPRHELIA Pharma, will attend this event. If you would like to meet with Hugues and Jeremy, please send an email to: contact@orphelia-pharma.eu.
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.
We are glad to announce that Caroline Lemarchand, Chief Pharmaceutical Development Officer at ORPHELIA Pharma, will attend this event and present the following session: “Formulation and assessment of Kimozo® an oral liquid formulation of temozolomide usable in children as young as 1 year old”. If you would like to meet Caroline, please contact her : caroline.lemarchand@orphelia-pharm.eu.