On December 10, 2019 ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric drugs in the fields of oncology and neurology, reported that the Office for Orphan Products of the Food and Drug Administration (FDA) has issued a positive opinion for the Orphan Drug Designation of temozolomide in the treatment of neuroblastoma (Press release, ORPHELIA Pharma, DEC 10, 2019, View Source [SID1234552180]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
« We are particularly pleased with the grant of this Orphan Designation », comments Jérémy Bastid, Chief Development Officer of ORPHELIA Pharma. « Temozolomide is an important part of the treatment armamentarium for high-risk neuroblastoma patients who relapse or face disease recurrence, albeit used off‑label. Existing formulations of temozolomide-containing products are not adapted to young children. Our formulation Kimozo aims at providing these young patients with a pharmaceutical form adapted to their age », he concludes.
Kimozo is the first presentation of the anticancer drug temozolomide developed for the treatment of relapsed or refractory neuroblastoma, a pediatric disease affecting young patients with dismal prognosis. Kimozo is being developed as a taste-masked oral suspension and will be the first pediatric formulation of temozolomide.
« Temozolomide has become an integral component of established regimens for relapsed and refractory neuroblastoma. Patients with neuroblastoma are usually younger than 5 years of age and unable to tolerate the currently available temozolomide tablet formulations, presenting barriers to administration. The liquid formulation of Kimozo is therefore a welcome and needed resource for treating patients with neuroblastoma », highlights Dr. Julie Park, Department of Pediatrics, Seattle Children’s Hospital and University of Washington School of Medicine, Seattle.
« The Office for Orphan Products of the FDA has acknowledged the benefit of temozolomide in the treatment of neuroblastoma patients with relapsed of refractory disease. The pediatric 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. « We expect to file Kimozo Marketing Authorization Application with the FDA as soon as the dossier is complete ».
About temozolomide in neuroblastoma
Neuroblastoma is the most frequent extra-cranial solid tumor in children. It is a rare disease with an incidence of 1.3/100,000. Prognosis is extremely variable, from spontaneously regressing tumors 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.