IDM Pharma, Inc. has announced that it has recently met with the U.S. Food and Drug Administration (FDA) and informed the FDA that the Company intends to take immediate action to supplement the data in its current New Drug Application (NDA) for mifamurtide (L-MTP-PE), formerly known as Junovan, which is being reviewed for the treatment of children and adolescents with non-metastatic osteosarcoma.
The Company plans to collect and submit additional Phase 3 data that will support the benefit of L-MTP-PE in the treatment of non-metastatic osteosarcoma. Specifically, the Company will continue working with the cooperative groups and investigative sites involved in the study to collect vital status (information on whether the subjects remain alive or have died) on patients who participated in the Phase 3 clinical trial and for which data was not available at the time of filing of the NDA in October 2006. When the additional follow up data have been collected, the Company will analyze the data set and expects to submit an amendment to the NDA to the FDA by the first quarter of 2008. In addition to collecting supplemental Phase 3 data, the Company is also working on addressing other concerns raised at the Oncologic Drugs Advisory Committee (ODAC) meeting held in May 2007 and discussed in the recent meeting with the FDA.
"While we believe the overall survival benefit of L-MTP-PE has been demonstrated, we acknowledge the issues raised by the FDA's review of our NDA, in part based on the submitted data set," said Timothy P. Walbert, President and Chief Executive Officer of IDM Pharma, Inc. "We are confident that these efforts will allow a more robust analysis of L-MTP-PE, will continue to support its overall survival benefit in osteosarcoma and will provide substantial evidence for the potential regulatory approval of this important treatment."
L-MTP-PE stimulates the innate immune system, or the body's first line of defense, to kill tumor cells, and based on data from clinical studies, when used in combination with surgery and chemotherapy, L-MTP-PE reduces the risk of recurrence of osteosarcoma and improves long term survival.
The L-MTP-PE NDA includes efficacy and safety data from 678 patients with non-metastatic resectable osteosarcoma, 332 of whom received L-MTP-PE, and from 115 patients with metastatic or unresectable osteosarcoma, 39 of whom received L-MTP-PE, in the controlled Phase 3 trial conducted by the Pediatric Oncology Group (POG) and the Children's Cancer Group (CCG), now the Children's Oncology Group (COG), sponsored by the Cancer Therapy Evaluation Program (CTEP) of the National Cancer Institute. Also included are safety and efficacy data from 51 patients with metastatic osteosarcoma treated in earlier Phase 2 studies. The biological effects and safety of L-MTP-PE are further supported by data from 15 other Phase 1 and 2 clinical studies in which an additional 197 patients received at least one dose of L-MTP-PE.
L-MTP-PE stimulates the innate immune system (the body's first line of defense) to kill tumor cells. When administered in combination with chemotherapy and after tumor resection to osteosarcoma patients in the Phase 3 trial, L-MTP-PE provided a significant improvement in Disease Free Survival (DFS) (p = 0.0245) and Overall Survival (OS) (p = 0.0183). At 6 years, the probability of survival when L-MTP-PE is combined with adjuvant chemotherapy is 77% (95%CI: 72-83%) compared to 66% (95%CI: 59-73%) without L-MTP-PE, a clinically meaningful finding in a pediatric population where the longer the survival, the greater the chance the patient is cured of cancer.
Treatment with L-MTP-PE was generally well tolerated in all phases of study. 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.