On September 3, 2019 ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, reported 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 (Press release, ORPHELIA Pharma, SEP 3, 2019, View Source [SID1234539181]).
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« 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.