IDM Pharma, Inc. has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) and the recommendation to grant centralized marketing authorization for mifamurtide (L-MTP-PE), known as MEPACT(R) in Europe, for the treatment of patients with non-metastatic, resectable osteosarcoma. Final approval from the European Commission is expected in early 2009 once the recommendation has been adopted at the next CHMP meeting.

Once authorized, the product can be marketed in the European Union (EU), as well as in Iceland, Liechtenstein and Norway. LMTP- PE would be the first approved new treatment in over 20 years for patients with osteosarcoma, a rare and often fatal bone tumor typically affecting children and young adults. LMTP- PE was granted orphan medicinal product status in Europe in 2004, and as a result, is entitled to a period of 10 years market exclusivity.

CHMP’s positive opinion was based on the phase III L-MTP-PE trial (INT-0133), a cooperative group study conducted by the Children's Oncology Group (COG) and funded by the National Cancer Institute (NCI). To date the largest study completed in osteosarcoma, it enrolled approximately 800 patients and evaluated patient outcomes with the addition of L-MTP-PE to three- or four-drug adjuvant chemotherapy.

After six years of follow-up in patients treated with chemotherapy and L-MTP-PE, overall survival was 78 percent, compared to 70 percent in patients treated with chemotherapy alone. The addition of L-MTP-PE to chemotherapy resulted in approximately a 30% decrease in the risk of death. The treatment was generally well tolerated in all phases of clinical development, with mild to moderate adverse events, consistent with the activation of monocytes and macrophages by L-MTP-PE and the flu-like symptoms that follow cytokine release; easily prevented or treated with acetaminophen.

If approved by the EMEA, the company would address follow up questions about L-MTP-PE by conducting post-authorization studies or analyses. The company continues to work in the US to respond to the non-approvable letter received from the U.S. Food and Drug Administration (FDA) and expects to submit the amended New Drug Application (NDA) in the first quarter of 2009. 

L-MTP-PE was granted orphan drug status in the United States in 2001 and the NDA was submitted to FDA in October 2006 and was accepted for review in December 2006.

IDM Pharma is a biopharmaceutical company focused on developing cancer therapies by activating the immune system.

Source: IDM Pharma, Inc.