In March 2009, the European Commission formally granted a centralized marketing authorization for IDM Pharma's Mepact® (mifamurtide, L-MTP-PE) for the treatment of patients with osteosarcoma (malignant bone cancer). Specifically, mifamurtide is indicated for the treatment of high-grade, resectable, nonmetastatic osteosarcoma after complete surgical resection of the tumor in children, adolescents and young adults between the ages of 2 and 30. Mifamurtide is indicated for use in combination with other anticancer medicines after the cancer has been removed by surgery. The centralized marketing authorization allows mifamurtide to be marketed in the 27 member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

Osteosarcoma is a rare and often fatal disease, with approximately 1,200 new cases diagnosed in Europe each year, primarily children and young adults. The standard treatment for osteosarcoma is surgical removal of the tumor (resection) with combination chemotherapy before and after surgery.

Mifamurtide is a liposomal formulation of MTP-PE (muramyl tripeptide phosphatidylethanolamine) designed specifically to target and activate macrophages. It is a fully synthetic derivative of muramyl dipeptide, a naturally occurring component of bacterial cell walls, for administration by intravenous infusion. The approval was based on a phase III trial, a National Cancer Institute (NCI)-funded cooperative group study conducted by the Children's Oncology Group, enrolling approximately 800 patients. Results demonstrated that the addition of mifamurtide to chemotherapy resulted in approximately a 30% decrease in the risk of death, with 78% of patients surviving through the sixth year of follow-up after treatment (Meyers, P.A. et al., J Clin Oncol 2008, 26(4): 633-8). Mifamurtide has orphan drug status in the E.U. for this indication.

Subsequent to the approval, IDM Pharma was acquired by Takeda, which began launching Mepact® in February 2010. Mifamurtide is the first new treatment in 20 years to improve survival in patients with osteosarcoma. The treatment will be available via a paid named-patient program in countries where it is not initially reimbursed.