IDM Pharma, Inc announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) has issued a positive opinion, recommending grant of a centralized marketing authorization for mifamurtide (L-MTP-PE), known as Mepact in Europe, 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 CHMP recommendation will be adopted at the next CHMP meeting in December with final European Commission approval expected within 60 to 90 days thereafter.
Granting of the centralized marketing authorization will allow L-MTP-PE to be marketed in the 27 Member States of the European Union (EU), as well as in Iceland, Liechtenstein and Norway. L-MTP-PE would be the first approved new treatment in more than 20 years for patients with osteosarcoma. L-MTP-PE was granted orphan medicinal product status in Europe in 2004. Therefore, under European pharmaceutical legislation, L-MTP-PE is entitled to a period of 10 years market exclusivity in respect of the approved indication.
"The recommendation for approval by the CHMP is a great victory for many young patients and their families and is a significant step for the company in bringing this important treatment to market," said Timothy P Walbert, president and chief executive officer, IDM Pharma. "The committee's decision validates the clinical trial data and the belief of investigators, patients and IDM Pharma that L-MTP-PE provides a significant overall survival benefit for osteosarcoma patients and meets a significant unmet treatment need."
The positive opinion was based in large part on the phase-3 L-MTP-PE trial (INT-0133), a National Cancer Institute (NCI) funded cooperative group study conducted by the Children's Oncology Group (COG) and the largest study completed in osteosarcoma, enrolling approximately 800 patients. The study was designed to evaluate patient outcomes with the addition of L-MTP-PE to three- or four-drug adjuvant chemotherapy (cisplatin, doxorubicin, and methotrexate with or without ifosfamide).
Overall survival after six years of follow-up in patients treated with chemotherapy and L-MTP-PE was 78 per cent, compared to 70 per cent in patients treated with chemotherapy (p=0.03) alone. The addition of L-MTP-PE to chemotherapy resulted in approximately a 30 per cent decrease in the risk of death.
Treatment with L-MTP-PE was generally well tolerated in all phases of clinical development. Adverse events were mild to moderate in severity and included chills, fever, nausea, vomiting, myalgia, headache, tachycardia (fast heart rate), hypo- and hypertension, fatigue and shortness of breath, all of which are consistent events with the activation of monocytes and macrophages by L-MTP-PE and the flu-like symptoms that follow cytokine release. These side effects are readily prevented or treated with acetaminophen.
If approved by the European Commission, it is anticipated the company would conduct certain post-authorization studies or analyses to address follow up questions about L-MTP-PE.
As previously announced, in the United States 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 L-MTP-PE and to respond to other questions in the non-approvable letter the company received from the US Food and Drug Administration (FDA). The company expects to submit the amended New Drug Application (NDA) in the first quarter of 2009 given the recent focus on completing review activities for the Marketing Authorization Application (MAA) in the European Union.
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 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.