IDM Pharma has submitted an electronic New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Junovan (mifamurtide for injection), requesting approval for its use in the treatment of newly diagnosed resectable high grade osteosarcoma patients following surgical resection in combination with multiple agent chemotherapy.
The Junovan NDA submission includes efficacy and safety data from 678 patients with non-metastatic resectable osteosarcoma, 332 of whom received Junovan, and from 115 patients with metastatic or unresectable osteosarcoma, 39 of whom received Junovan in the controlled phase III trial conducted by the Pediatric Oncology Group (POG) and the Children's Oncology Group (COG), sponsored by the division of cancer treatment and diagnosis of the National Cancer Institute (NCI). The biological effects and safety of Junovan are further supported by data from 17 phase I and II clinical studies performed by Ciba-Geigy in which an additional 248 patients received at least one dose of Junovan.
Junovan (Mifamurtide - Liposomal Muramyl Tripeptide Phosphatidyl Ethanolamine, MTP-PE) is a fully synthetic lipophilic derivative of the muramyl dipeptide. When encapsulated in liposomes, MTP-PE is delivered selectively to macrophages via the scavenger lipoprotein receptor pathway. When the multilamellar liposomes are degraded inside the macrophage, MTP-PE is released, activating tumouricidal activity through the cytoplasmic Nod2 receptor. The product was originally developed by Ciba-Geigy and IDM completed the development of the product manufacturing and performed the required analyses including the Phase III analyses for the FDA submission.
Jean-Loup Romet-Lemonne, M.D., president and CEO of IDM, stated that, "This NDA filing represents new hope for survival for patients with osteosarcoma. This is a bone cancer that generally metastasizes to the lung and affects mainly children and young adults. At IDM, we are very pleased to submit Junovan to the FDA with the hope of improving the outcome of these young patients for whom very few new therapeutic options have been made available over the last two decades."
Dr. Paul Meyers, vice chair of Pediatrics at Memorial Sloan-Kettering Cancer Centre and principal investigator for the phase III trial, said, "Osteosarcoma is a childhood cancer for which the outcome, despite advances in surgical and chemotherapeutic approaches, can be very poor in a substantial proportion of patients. Mifamurtide, if made commercially available, will provide treating physicians with a new tool with significant potential to improve patient outcome and, most importantly, survival."
IDM requested that the FDA consider granting this NDA priority review status, which could shorten review time from the standard ten months to six months if granted. The FDA customarily accepts or refuses to file NDAs and designates review status within 60 days of filing. The FDA and the European Medicines Evaluation Agency (EMEA) have designated orphan drug status to Junovan.
About 3 per cent of all childhood cancers are osteosarcoma. Because osteosarcoma usually develops from osteoblasts, it most commonly develops in teenagers who are experiencing their adolescent growth spurt. Osteosarcoma is an orphan disease and there are approximately 1000 new cases in the US each year. A similar incidence of the disease exists in Europe. According to the children's oncology group.
The survival of children with osteosarcoma has remained at 60-65 per cent since the mid-1980s. The standard treatment for osteosarcoma is tumor resection with combination chemotherapy before and after surgery.