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.
We would be glad to meet you there and discuss partnering opportunities with ORPHELIA Pharma. Please contact us through the partnering portal at: https://informaconnect.com/bioeurope/pone/login/
Première formulation buvable de témozolomide pour le traitement des neuroblastomes réfractaires ou en rechute
Paris et Lyon, France, le 24 octobre 2023 – Orphelia Pharma, société pharmaceutique dédiée au développement et à la commercialisation de médicaments pédiatriques et orphelins, annonce aujourd’hui avoir déposé une demande d’autorisation de mise sur le marché (AMM) centralisée auprès de l’agence européenne du médicament (European Medicines Agency – EMA) pour KIZFIZO®, la première formulation orale liquide de témozolomide.
KIZFIZO (suspension buvable de témozolomide, 40 mg/ml), connu sous les noms de Ped-TMZ ou KIMOZO pendant son développement clinique et les programmes d’accès précoce en cours, a été conçu spécifiquement pour une utilisation dans le traitement des enfants atteints de neuroblastome récidivant ou réfractaire, indications d’oncologie de mauvais pronostic. Cette suspension buvable, dont le goût est masqué, a été développée pour les enfants : elle permet d’administrer une dose précise dans un petit volume par voie orale ou à l’aide d’une sonde nasogastrique. Orphelia Pharma développe KIZFIZO depuis six ans en collaboration avec Gustave Roussy, premier centre de cancérologie européen.
« Nous sommes très fiers d’avoir développé ce nouveau médicament pédiatrique », déclare Laurent Martin, directeur des affaires pharmaceutiques chez Orphelia Pharma. « KIZFIZO répond à un besoin médical non satisfait : disposer d’une suspension buvable de témozolomide pour le traitement du neuroblastome récidivant ou réfractaire. Cette formulation vise à éviter les mésusages qui consistent à utiliser des formes pharmaceutiques non adaptées à l’enfant mélangées à une boisson ou de la nourriture, ce qui peut exposer les aidants à une molécule cytotoxique et sans vrai contrôle de la dose effectivement délivrée au patient. »
« La demande d’autorisation de mise sur le marché de KIZFIZO est une excellente nouvelle pour les enfants vivant avec un cancer et leur famille. Elle souligne l’engagement de Gustave Roussy à jouer un rôle de premier plan dans le développement de médicaments pédiatriques dans l’objectif de guérir tous les enfants dans le futur », déclare le Professeur Fabrice Barlesi, Directeur Général de Gustave Roussy.
La pharmacocinétique de KIZFIZO chez les enfants a été évaluée dans TEMOkids, une étude multicentrique européenne de pharmacocinétique, d’acceptabilité et de tolérance chez le patient pédiatrique ayant besoin de témozolomide (NCT04610736).
Les données sur l’efficacité et l’innocuité du témozolomide dans le neuroblastome réfractaire ou en rechute, présentées dans la demande d’AMM, comprennent notamment :
BEACON-Chemo, une sous-analyse des bras de chimiothérapie de l’étude BEACON, étude prospective, randomisée, multicentrique européenne de phase II chez les enfants atteints d’un neuroblastome réfractaire ou récidivant. Le sponsor de cette étude est l’Université de Birmingham (Royaume-Uni).
Retro-TMZ, une étude descriptive et rétrospective multicentrique européenne, évaluant l’efficacité et la tolérance du témozolomide chez les enfants atteints d’un neuroblastome réfractaire ou récidivant. Cette étude a été réalisée par Gustave Roussy (France).
À propos de KIZFIZO® 40 mg/ml KIZFIZO (témozolomide suspension buvable, 40 mg/ml) est une formulation pédiatrique liquide de témozolomide pour administration par voie orale, prête à l’emploi, développée pour le traitement du neuroblastome récidivant ou réfractaire. Cette formulation, au goût masqué et adapté aux jeunes enfants, permet une administration précise dans un petit volume tout en évitant la manipulation du produit et l’exposition des aidants au témozolomide, médicament cytotoxique. Il est le fruit d’une collaboration entre les pharmaciens et cliniciens de Gustave Roussy et l’équipe de développement d’Orphelia Pharma. En mars 2022, KIZFIZO a obtenu une Autorisation d’Accès Précoce (AAP) par les autorités françaises, pour le traitement du neuroblastome réfractaire ou récidivant en monothérapie ou en association avec l’irinotécan ou le topotécan. KIZFIZO a obtenu une désignation de médicament orphelin (Orphan Drug Designation – ODD) auprès de l’EMA et de la FDA. La formulation est couverte par des brevets délivrés et des demandes en instance en Europe et aux États-Unis.
À propos du neuroblastome Le neuroblastome est un cancer extra-cérébral rare, avec environ 900 nouveaux cas diagnostiqués chaque année dans l’Union Européenne. Il touche presque exclusivement les enfants de moins de cinq ans, avec un âge médian de 18 mois au moment du diagnostic. Le neuroblastome a une grande diversité de pronostic, ce qui se reflète dans la stratification du risque. Environ 40% des patients présentent un neuroblastome de haut risque et font souvent face à une mauvaise réponse au traitement d’induction (patients réfractaires) ou à une rechute ultérieure. Il subsiste un réel besoin médical non satisfait pour ces patients et des recherches sont en cours pour déterminer la meilleure stratégie thérapeutique. Le témozolomide est la chimiothérapie couramment utilisée et constitue une partie essentielle de l’arsenal thérapeutique pour ces patients.
À propos d’Orphelia Pharma Orphelia Pharma est une société pharmaceutique basée à Paris et Lyon qui développe et commercialise des médicaments pour le traitement des maladies pédiatriques et orphelines. Elle fournit aux patients des produits essentiels dans les domaines de la neurologie et de l’oncologie, dans des formulations adaptées à la population pédiatrique. Orphelia Pharma mène des projets de recherche dans le cadre de partenariats académiques et industriels. Elle a récemment mis en place des accords régionaux dans les territoires européens et étend sa présence à travers le monde. www.orphelia-pharma.eu
A propos de Gustave Roussy Classé premier centre de cancérologie français, premier européen et troisième au niveau mondial, Gustave Roussy constitue un pôle d’expertise globale entièrement dédié aux patients vivant avec un cancer. L’Institut est un pilier fondateur du biocluster en oncologie Paris-Saclay Cancer Cluster. Source d’innovations thérapeutiques et d’avancées diagnostiques, l’Institut accueille chaque année près de 50 000 patients dont 3 500 enfants et adolescents et développe une approche intégrée entre recherche, soins et enseignement. Expert des cancers rares et des tumeurs complexes, Gustave Roussy traite tous les cancers, à tous les âges de la vie. Il propose à ses patients une prise en charge personnalisée qui allie innovation et humanité, où sont pris en compte le soin mais aussi la qualité de vie physique, psychologique et sociale. Avec 4 100 salariés répartis sur deux sites, Villejuif et Chevilly-Larue, Gustave Roussy réunit les expertises indispensables à une recherche de haut niveau en cancérologie ; 40% des patients traités sont inclus dans des études cliniques. Pour en savoir plus sur Gustave Roussy et suivre les actualités de l’Institut : www.gustaveroussy.fr, Twitter, Facebook, LinkedIn, Instagram
First drinkable formulation of temozolomide for treatment of relapsed or refractory neuroblastoma
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.
KIZFIZO (temozolomide oral suspension, 40 mg/ml), known as Ped-TMZ or KIMOZO during its clinical development and ongoing early access programs, is designed specifically for use in the treatment of children with relapsed or refractory neuroblastoma, oncology indications with a very poor prognosis. This oral suspension, which is taste-masked, was developed for children : it allows a precise dose to be administered orally or via a nasogastric tube in a small volume. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last six years.
“We take great pride in having developed this new pediatric medicine,” said Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma. “KIZFIZO fills an unmet medical need for a drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma. This formulation aims to avoid the use of non-age-appropriate dosage forms mixed with a drink or food, which may expose caregivers to a cytotoxic molecule without full control of the dose actually delivered to the patient.”
“The application for a drug marketing authorization of KIZFIZO is excellent news for the children living with cancer and their families. It underscores Gustave Roussy’s commitment to playing a leading role in the development of medicines for children and curing all of them in the future,” said Prof. Fabrice Barlesi, CEO of Gustave Roussy. The pharmacokinetics of KIZFIZO in children have been evaluated in TEMOkids, a European multicenter population pharmacokinetic acceptability and safety study in pediatric patients in need of temozolomide (NCT04610736).
Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma submitted in the application includes in particular :
BEACON-Chemo, a sub-analysis of the chemotherapy arms of the BEACON study, a prospective randomized phase II study in refractory or relapsed neuroblastoma. This study was sponsored by Birmingham University (UK)
Retro-TMZ, a multicenter descriptive, retrospective study, assessing the efficacy and tolerability of temozolomide in children with refractory or relapsed neuroblastoma. This study was conducted by Gustave Roussy (France)
About KIZFIZO® 40 mg/ml
KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma. In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with irinotecan or topotecan. KIZFIZO has received Orphan Drug Designation (ODD) from the EMA and the FDA, the formulation is covered by granted patents and pending applications in Europe and the US.
About neuroblastoma Neuroblastoma is the most common extracranial cancer in early childhood, with approximately 900 new cases diagnosed per year in the European Union. It almost exclusively affects children under five, with a median age at diagnosis of 18 months. Neuroblastoma has a wide diversity of clinical outcomes, which is reflected in the risk stratification. Approximately 40% of patients have the high-risk disease and often face a poor response to first line induction therapy or later relapse. There remains a high unmet need for relapsed or refractory neuroblastoma patients and the best therapeutic strategy is still an intensive area of research. Temozolomide is the standard chemotherapy and is therefore an essential part of the treatment armamentarium for these patients.
About Orphelia Pharma Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint across the world. www.orphelia-pharma.eu
About Gustave Roussy Ranked as the leading European Cancer Center and third in the world, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering from cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. It is a source of diagnostic and therapeutic advances. Per year, it caters for almost 50,000 patients, including 3,500 pediatric patients, 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. 4,100 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 : 40% of patients treated are included in clinical studies. For further information : www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram
Press and Analyst Contacts Andrew Lloyd and Associates Emilie Chouinard / Saffiyah Khalique emilie@ala.associates / saffiyah@ala.associates UK +44 1273 952 481 US +1 203 724 5950
Stop by our booth in the exhibition and poster area to learn more about our pipeline including KIZFIZO, temozolomide oral suspension (product in development).
You are also invited to attend the oral session entitled “novel therapeutic approaches” on Saturday morning. Dr. Lucy METAYER, pediatric oncologist at Gustave Roussy, will present the results of the TEMOkids pediatric trial.
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 55thSociété 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).
TEMOkids (NCT04610736) is a European, multicenter, population pharmacokinetic, acceptability and safety clinical study evaluating KIZFIZO® (temozolomide oral suspension, 40 mg/ml) in children from the age of one year and in need of temozolomide. KIZFIZO, formerly known as KIMOZO or Ped-TMZ, has been designed specifically for use in the treatment of children with relapsed or refractory neuroblastoma, an oncology indication of dismal prognosis.
“Forty-three young patients were recruited in TEMOkids across 12 European clinical cancer centers”, comments Caroline LEMARCHAND, Chief Pharmaceutical Development Officer at Orphelia Pharma. “TEMOkids is an integral part of the clinical development plan of KIZFIZO and results will be presented at the SIOP conference for the first time”.
“Results from TEMOkids are very compelling” adds Dr. Samuel ABBOU, principal investigator of theTEMOkids study and pediatric oncologist at Gustave Roussy. “Not only the population pharmacokinetics show that there is no need for temozolomide dose adjustment in the pediatric population, but KIZFIZO was well accepted by children and its safety profile was similar to that of temozolomide capsules (Temodal), with no specific signal of local intolerance”.
About the TEMOkids study (NCT04610736)
TEMOkids is an international, open-label, non-randomized, study evaluating Ped-TMZ oral suspension (KIZFIZO) in 40 pediatric patients aged 1 year and over. The main objective of this study was to determine the pharmacokinetic parameters of KIZFIZO in this population. The secondary objectives were to evaluate its tolerance and acceptability by children, and their response to treatment.
Twelve clinical centers were involved in the TEMOkids study: Gustave Roussy, Villejuif, France (coordinating center); Institut Curie, Paris, France; La Timone Children’s Hospital, Marseille, France; Institute of Pediatric Hematology and Oncology, Lyon, France; Oscar Lambret Center, Lille, France; Charité University Medical Hospital, Berlin, Germany; Princess Máxima 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, the United Kingdom and Great Ormond Street Hospital for Children NHS Trust, London, the United Kingdom.
About KIZFIZO 40 mg/ml
KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory neuroblastoma, the most common extracranial solid tumor of childhood carrying a dismal prognosis. This age-adapted and taste-masked formulation delivers an accurate drug load in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a fruitful collaboration between the pharmacists and clinicians of Gustave Roussy Cancer Center and the development team of Orphelia Pharma.
KIZFIZO, under the product name KIMOZO, has been granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities in March 2022 for the treatment of refractory and relapsed neuroblastoma.
KIZFIZO received orphan drug designation from the EMA and FDA and its formulation is covered by granted patents and pending applications in Europe and in the US.
About Orphelia Pharma
Orphelia Pharma is a pharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. The company’s mission is to provide patients with essential products in the fields of neurology and oncology, in formulations adapted to the pediatric population. Orphelia Pharma conducts research projects through academic and industrial partnerships. It has recently established regional agreements in European territories and is expanding its footprint on other continents.
Ranked as the leading European Cancer Centre and third in the world, Gustave Roussy is a centre with comprehensive expertise and is devoted entirely to patients suffering with cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. 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. 4,100 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: 40% of patients treated are included in clinical studies.
The SIOP Annual Meetings bring together more than 2,500 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. This congress represents a unique opportunity to stay up to date in this rapidly moving field and to bring the latest science to bedside. The 55th annual meeting will be held in Ottawa, Canada, October 11-14, 2023.
