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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

9 June 2022

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.