ORPHELIA at the 22nd French Epilepsy Days
28 août 2019
ORPHELIA at the XIIth National Oncology Days of the French Society of Pharmacy Oncology
9 septembre 2019

Orphan Drug Designation granted for temozolomide in the treatment of neuroblastoma

ORPHELIA Pharma announces Orphan Drug Designation granted by EMA for temozolomide in the treatment of neuroblastoma

Development of Kimozo®, the first paediatric formulation of temozolomide

 Paris and Lyon, September 3rd, 2019 – 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 the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion for the Orphan Drug Designation of temozolomide in the treatment of neuroblastoma.

« We are particularly pleased with this decision which comes after continuous efforts of ORPHELIA Pharma together with its partners », comments Jérémy Bastid, Chief Development Officer. « We are developing Kimozo®, the first paediatric formulation of temozolomide, to provide patients and the medical community with a pharmaceutical form adapted to young children. This positive opinion is one step closer to this goal ».

Kimozo® is the first presentation of the anticancer drug temozolomide developed for the treatment of relapsed or refractory neuroblastoma, a paediatric disease with dismal prognosis. As a taste-masked oral suspension, it will also be the first paediatric formulation of temozolomide.

« Temozolomide has been used for years in the treatment of refractory or relapsed neuroblastoma. It is now considered to be an essential drug in this indication », highlights Dominique Valteau-Couanet, President of the Société Internationale d’Oncologie Pédiatrique, Europe Neuroblastoma (SIOPEN), « The liquid formulation of Kimozo®, which will be the only temozolomide-containing drug approved for the treatment of neuroblastoma, is particularly well adapted to the intended paediatric population since 90% of the patients are less than 5 years at diagnosis ».

« The COMP has acknowledged the benefit of temozolomide in the treatment of children with high-risk neuroblastoma. The paediatric formulation that we are developing addresses significant unmet medical needs for young children with neuroblastoma », comments Hugues Bienaymé, Founder and General Manager of ORPHELIA Pharma. « In Europe, we expect to submit Kimozo® Marketing Authorisation application file by 2021 ».

About temozolomide in neuroblastoma

Neuroblastoma is the most frequent extra-cranial solid tumour in children. It is a rare disease with an incidence of 1.3/100,000. Prognosis is extremely variable, from spontaneously regressing tumours in low-risk neuroblastomas to highly aggressive disease with dismal prognosis in high-risk patients. Half of neuroblastomas are classified as high-risk, among which half being refractory to treatment or relapsing thereafter. Temozolomide has become the mainstay of rescue treatment of relapsed or refractory neuroblastoma, without being authorized in this condition.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of rare and serious paediatric diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, obtained its Marketing Authorization in October 2018 and has been launched in Europe. Our second product, Ivozall®, is under review by the EMA for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma also conducts research projects through academic and industrial collaborations. The main investors are initiative OCTALFA and Pierre Fabre Medicament.