IDM Pharma, Inc announced that the European Commission has formally granted a centralized marketing authorization for Mepact (mifamurtide, L-MTP-PE) for the treatment of patients with non-metastatic, resectable osteosarcoma, a rare and often fatal bone tumour that typically affects children and young adults.
The centralized marketing authorization allows Mepact to be marketed in the 27 Member States of the European Union, as well as in Iceland, Liechtenstein and Norway. Mepact was granted orphan medicinal product status in Europe in 2004 and under European pharmaceutical legislation is entitled to a period of 10 years market exclusivity in respect of the approved indication.
"Today's approval of Mepact is a significant milestone for physicians and patients in Europe, giving them access to the first new osteosarcoma treatment option in 20 years," said Timothy P Walbert, president and chief executive officer, IDM Pharma. "As our lead product candidate and first to receive approval, this is also a major milestone for IDM Pharma. We look forward to amending the New Drug Application (NDA) for mifamurtide in the United States and continuing to work toward bringing this important treatment to market in the US."
The approval was based on the phase-3 Mepact trial (INT-0133), a National Cancer Institute (NCI) funded cooperative group study conducted by the Children's Oncology Group (COG) and the largest study ever completed in osteosarcoma, enrolling approximately 800 patients. The study evaluated patient outcomes with the addition of Mepact to three- or four-drug adjuvant chemotherapy (cisplatin, doxorubicin, and methotrexate with or without ifosfamide). Results demonstrated that the addition of Mepact to chemotherapy resulted in approximately a 30 per cent decrease in the risk of death with 78 per cent of patients surviving after six years of follow-up after treatment with Mepact.
"Mepact is the first therapy in more than 20 years to demonstrate any significant long-term survival advantage in osteosarcoma," said Ian Lewis, professor of Cancer Research at St James University Hospital in Leeds, England. "The approval of Mepact is the culmination of two decades of research and dedication to children and young adults with osteosarcoma and brings real hope for a patient population in need of an innovative treatment option for this devastating disease."
"As an investigator who has been involved in the development of Mepact, I am thrilled that years of hard work and commitment by researchers around the world has resulted in this positive outcome," said Eugenie Kleinerman, professor and head of the Division of Paediatrics and professor of Cancer Biology at The University of Texas Maryland Anderson Cancer Center. "This is a remarkable advance for treatment of young patients with osteosarcoma and should give physicians and their patients hope in treating this rare disease."
The company continues to evaluate strategic alternatives, which may include seeking strategic partners, a merger and/or the sale of all or part of its operations and assets, or raising additional capital to secure operational sales and marketing infrastructure for Mepact.
As previously announced, in the US, the company continues to work with the COG as well as external experts and advisors to gather patient follow up data from the phase-3 clinical trial of mifamurtide and to respond to other questions in the non-approvable letter the company received from the US Food and Drug Administration (FDA). The company plans to submit an amended New Drug Application (NDA) for mifamurtide in mid-2009 and expects to be in a position to provide an update on the progress of the filing, including timing, following a meeting scheduled with the FDA in March.
Mifamurtide was granted orphan drug status in the United States in 2001. The NDA was submitted to FDA in October 2006 and was accepted for review in December 2006.
Between two and three percent of all childhood cancers are osteosarcoma. Because osteosarcoma usually develops from osteoblasts, it most commonly affects children and young adults experiencing their adolescent growth spurt. Boys and girls have a similar incidence rate until later in their adolescence, when boys are more commonly affected. While most tumours occur in larger bones, such as the femur, tibia, and humerus, and in the area of the bone that has the fastest growth rate, they can occur in any bone. The most common symptom is pain, but swelling and limited movement can occur as the tumour grows.
IDM Pharma is focused on the development of innovative cancer products that either destroy cancer cells by activating the immune system or prevent tumour recurrence by triggering a specific adaptive immune response.