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/).

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).

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.

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 : orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

Tanner Pharma Group : lstevens@tannerpharma.com / Tel: +1 704 552 8408

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.

« 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 : orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

ORPHELIA Pharma announces completion of patient recruitment for 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.

“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 : orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

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.

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

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.

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/).

SIOP 2022, Barcelona, Spain : September 28-October 1, 2022

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.

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.

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.

Event: Organisation Inter Régionale Hémato-Onco pédiatrique (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. This first annual meeting aims to gather doctors, researchers, clinicians, paramedics, from the EN-HOPE inter-region aims and to facilitate collaborations. Jeremy Bastid, Chief Operating Officer at ORPHELIA Pharma, will attend the event. If you would like to organize a meeting with Jeremy, please contact him : jeremy.bastid@orphelia-pharma.eu.

1ère journée de l’Organisation Inter Régionale Hémato-Onco pédiatrique (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).

Ce premier rendez-vous annuel pour les médecins, chercheurs, cliniciens, personnels paramédicaux, de l’inter région EN-HOPE a pour objectifs de favoriser le partage d’expertise dans différents domaines: médical, paramédical, sciences humaines et sociales, ainsi que la mise en place de réseaux et de projets collaboratifs multicentriques.

Jeremy Bastid, Directeur du Développement d’ORPHELIA pharma, participera à cet évènement. Vous pourrez rencontrer Jeremy sur le stand d’ORPHELIA Pharma. Vous pouvez également le contacter pour organiser un rdv: jeremy.bastid@orphelia-pharma.eu.

OCTALFA announces inclusion of the first patient in the French EAP of KIMOZO

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.

KIMOZO® 40 mg/ml (oral suspension of temozolomide) was granted an early access pre-marketing authorization (Autorisation d’Accès Précoce, AAP) in France on March 31st, 2022 by the Haute Autorité de Santé (HAS) for the treatment of relapsed or refractory neuroblastoma in patients aged 1 to 6 years as well as patients unable to swallow temozolomide capsules.

The goal of Early Access Programs in France is to accelerate access to innovative drugs before or after Market Authorization is granted once all conditions specified in Article L.5121-12 of the French Public Health Code (Code de la Santé Publique) are met:

  • No appropriate treatment exists;
  • The treatment’s implementation cannot be postponed;
  • The efficacy and safety of these drugs are strongly presumed based on therapeutic trials;
  • These drugs are considered to be innovative, particularly when compared to a possible clinically-relevant comparator.

Full details of the early access program can be found at the Haute Autorité de Santé and the ANSM websites.

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed to treat rare pediatric cancers.

In France, KIMOZO® has been granted an Early Access Program, to be used as monotherapy or in combination with irinotecan or topotecan in the treatment of pediatric patients aged 1 to 6 years and in patients with incapacity to swallow capsules of temozolomide and who suffer from:

  • refractory high-risk neuroblastoma or presenting an insufficient response to induction chemotherapy;
  • relapsed high-risk neuroblastoma after at least a partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.

KIMOZO® was developed by ORPHELIA Pharma in collaboration with Gustave Roussy.

About ORPHELIA Pharma

Founded by Hugues Bienaymé, ORPHELIA Pharma is a biopharmaceutical company based out of Paris and Lyon which develops and markets drugs for the treatment of pediatric and rare diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the rare diseases therapeutic area, in the form of formulations adapted to a pediatric population. With two drugs approved in the European Union, and another investigational medicine in a late stage of clinical development, ORPHELIA Pharma has recently established regional agreements in Europe, the United States and China, and is conducting research projects through academic and industrial partnerships.

For more information, please visit www.orphelia-pharma.eu

About OCTALFA

Set up by Gilles Alberici, OCTALFA is an independent family-owned company that has been developing and innovating for 15 years in the fields of life sciences, biotechnology and healthcare. Thanks to our subsidiaries and participations, we assert our presence in branches of medicines, particularly in pediatrics, cancer immunology, diagnostics and nanomaterials. We also provide support to patients living with cancer and people with disabilities, particularly children, in France and Madagascar, thanks to our endowment fund and the Dominique & Tom Alberici – OCTALFA Corporate Foundation, whose actions have been fully integrated into OCTALFA’s governance strategy since 2008.

OCTALFA is a shareholder of ORPHELIA Pharma since 2015.

For more information, please visit initiative-octalfa.eu

Contacts

Charlotte Maddalena / e-mail: charlotte.maddalena@octalfa.eu / phone: +33 (0)4 37 49 87 20

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

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).

« Depuis 2015, nous fabriquons une préparation hospitalière liquide de témozolomide pour répondre aux besoins des jeunes patients traités dans notre institut et quelques centres partenaires », explique Maxime Annereau, pharmacien à Gustave Roussy et à l’origine de ce projet. « En collaboration avec ORPHELIA Pharma, nous avons mené les travaux de recherche nécessaires pour optimiser les propriétés physico-chimiques et la stabilité de la suspension. Puis nous avons conduit une étude de palatabilité afin de sélectionner l’arôme capable de couvrir l’amertume du témozolomide de la manière la plus efficace. La mise au point de cette formulation concentrée a permis d’obtenir une forme pratique pour les soignants et les patients.», ajoute-t-il.

« Les travaux menés avec notre partenaire hospitalier Gustave Roussy ont conduit au prototype de KIMOZO®, version industrialisée et brevetée de cette suspension buvable concentrée. KIMOZO® est actuellement en phase avancée de développement clinique », indique Jeremy Bastid, Directeur du Développement d’ORPHELIA Pharma. « Le témozolomide est un médicament essentiel dans la prise en charge de nombreuses pathologies cancéreuses pédiatriques, et tout particulièrement du neuroblastome réfractaire ou en rechute qui touche malheureusement de très jeunes enfants. Grâce aux résultats de cette collaboration, nous entendons mettre rapidement cette forme pédiatrique à disposition des patients », conclut-il.

A propos de KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml, suspension pédiatrique buvable de témozolomide prête à l’emploi et avec masquage de goût, est le fruit d’une collaboration entre les pharmaciens et cliniciens de Gustave Roussy et les équipes de développement d’ORPHELIA Pharma. La formulation de KIMOZO a fait l’objet d’une demande de brevet en Europe et aux Etats-Unis.

A propos de Gustave Roussy

Classé premier centre européen et sixième mondial, Gustave Roussy constitue un pôle d’expertise globale entièrement dédié aux patients atteints de 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 près de 50 000 patients chaque année 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 ; un quart des patients traités sont inclus dans des essais cliniques.

Pour en savoir plus sur Gustave Roussy et suivre les actualités de l’Institut : www.gustaveroussy.fr, Twitter, Facebook, LinkedIn, Instagram

Contact : Claire Parisel / presse@gustaveroussy.fr / +33 (0)1 42 11 50 59

À 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.

Publication in Pharmaceuticals of the first temozolomide oral suspension

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).

“Since 2015, we have been manufacturing a liquid temozolomide hospital compounded formulation to address the needs of young patients treated in our hospital and a few partner centers,” explains Maxime Annereau, pharmacist at Gustave Roussy and the originator of this project. “In collaboration with ORPHELIA Pharma, we carried out the necessary research work to optimize the physico-chemical properties and the stability of the suspension.  Then we conducted a palatability study in order to select the aroma capable of covering the bitterness of temozolomide in the most efficient way. The development of this concentrated suspension has made it possible to obtain a form adapted to the needs of patients and caregivers”, he adds.

“The work done in collaboration with our partner Gustave Roussy led to a prototype of KIMOZO®, an industrialized and patented version of this drinkable and highly concentrated suspension. KIMOZO® is currently in an advanced phase of clinical development,” says Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “Temozolomide is an essential drug in the management of many pediatric cancers, including relapsed or refractory neuroblastoma which unfortunately affects very young children. Thanks to the results of this collaboration, we intend to make this pediatric formulation available to patients soon,” he concludes.

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed in collaboration between the pharmacists and clinicians of Gustave Roussy and the development team of ORPHELIA Pharma. The formulation of KIMOZO® has been covered by a patent application in Europe and in the US.

About Gustave Roussy

Classed as the leading European Cancer Centre and the sixth on the world stage, Gustave Roussy is a center 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: a quarter of patients treated are included in clinical trials.

For further information: www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram

Contact : Claire Parisel / presse@gustaveroussy.fr / +33 (0)1 42 11 50 59

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products with formulations adapted to the pediatric population. With two medicines approved in the European Union and one investigational medicine in an advanced clinical development phase, ORPHELIA Pharma has recently signed regional distribution agreements in the European Union, the USA and China with several partners and is carrying out research projects through academic and industrial partnerships.

Positive results of the bioequivalence study of Kimozo®

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.

The abstract #368704 entitled “bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary central nervous system malignancies“ by Ducray et al., was selected by the Scientific Program Committee of the 2022 ASCO annual meeting. The data released online demonstrate the equivalence between KIMOZO® oral suspension and temozolomide capsules.

“The positive results of the bioequivalence study are an important milestone in the development of KIMOZO®”, comments Caroline Lemarchand, Chief Pharmaceutical Development Officer at ORPHELIA Pharma. “KIMOZO® is the first drinkable form of temozolomide that has been specifically developed to address the needs of young children with cancer”, she adds.

“Temozolomide is part of the standard of care for the treatment of relapsed or refractory neuroblastoma, a devastating cancer that affects young children. KIMOZO® is currently undergoing further clinical evaluation (TEMOkids trial, NCT04610736) and our goal is to make this pediatric formulation of temozolomide available to children in the shortest timeframe possible”; concludes Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma.

About the bioequivalence study (NCT04467346)

The NCT04467346 bioequivalence study was an open label, randomized, crossover, 2-period study in 30 adult patients with primary CNS malignancies. The study objective was to evaluate primarily the bioequivalence between KIMOZO® oral suspension and TEMODAL® capsules, to define KIMOZO® pharmacokinetic parameters and to assess the safety and tolerability including buccal tolerance of KIMOZO®.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of neurology and oncology, with formulations adapted to the pediatric population. With two medicines approved in the European Union and one investigational medicine in an advanced clinical development phase, ORPHELIA Pharma has recently signed regional agreements in the European Union, the USA and China with several partners and is carrying out research projects through academic and industrial partnerships.

ORPHELIA annonce les résultats positifs de l’étude de bioéquivalence de KIMOZO® (suspension orale de témozolomide)

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.

La communication #368704 intitulée “bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary central nervous system malignancies“ (Ducray et al.), a été sélectionnée par le comité du programme scientifique du congrès annuel de l’ASCO 2022. Les données publiées en ligne démontrent l’équivalence de la suspension buvable KIMOZO® et des gélules de témozolomide.

« Les résultats positifs de cette étude de bioéquivalence constituent une étape importante dans le développement de KIMOZO® », commente Caroline Lemarchand, Directeur du Développement Pharmaceutique chez ORPHELIA Pharma. « KIMOZO® est la première forme buvable de témozolomide spécifiquement développée pour répondre aux besoins des jeunes enfants atteints de cancer », ajoute‑t-elle.

« Le témozolomide fait partie du traitement de référence du neuroblastome réfractaire ou en rechute, un cancer au pronostic sombre qui touche les jeunes enfants. KIMOZO® est actuellement en cours d’évaluation clinique (essai TEMOkids, NCT04610736) et notre objectif est de mettre cette formulation pédiatrique de témozolomide à la disposition des enfants dans les plus brefs délais » ; conclut Jeremy Bastid, Directeur du Développement d’ORPHELIA Pharma.

À propos de l’étude de bioéquivalence (NCT04467346)

L’étude de bioéquivalence NCT04467346 était une étude ouverte, randomisée, en cross-over, à 2 périodes menée chez 30 patients adultes atteints de tumeurs malignes primaires du système nerveux central. L’objectif de l’étude était de démontrer la bioéquivalence entre la suspension buvable KIMOZO® et les gélules de TEMODAL®, de définir les paramètres pharmacocinétiques de KIMOZO® et d’évaluer la sécurité et la tolérance, y compris la tolérance buccale, de KIMOZO®.

À 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 de la neurologie et de l’oncologie, 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.

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

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.

« Nous collaborons activement avec des équipes académiques à ce projet dont le développement clinique pourrait débuter d’ici 2 ans », indique Jérémy Bastid, Directeur du Développement d’ORPHELIA Pharma. « Il s’agit d’un projet ambitieux pour lequel nous disposons d’un rationnel scientifique solide et qui pourrait améliorer significativement la prise en charge de ces patients », ajoute-t-il.

« Nous remercions les équipes de Bpifrance pour leur soutien renouvelé qui nous permet d’amorcer le développement d’un médicament susceptible de devenir une nouvelle option thérapeutique dans cette indication orpheline » conclut Hugues Bienaymé, Directeur Général.

À 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 hospitaliers essentiels dans les domaines de la neurologie et de l’oncologie, 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.

€ 1 million grant awarded 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.

“We collaborate actively with academic teams on this program, the clinical development of which could begin within 2 years,” comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “This is an ambitious project for which we have a strong scientific rationale and that could improve the care of these patients“, he adds.

“We would like to thank the Bpifrance teams for their renewed support, which has enabled us to initiate the development of a drug which could become a new therapeutic option in this orphan indication,” concludes Hugues Bienaymé, General Manager.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential hospital products in the fields of neurology and oncology, with formulations adapted to the paediatric population. With two medicines approved in the European Union and one product in an advanced phase of clinical development, ORPHELIA Pharma has recently set up regional agreements in the European Union, the USA and China and is carrying out research projects through academic and industrial partnerships.

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

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

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.