Jul 23 2007
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.
The NDA for L-MTP-PE was submitted to the FDA in October 2006 and was accepted on December 26, 2006. L-MTP-PE was granted orphan drug status in the United States in 2001.
The FDA's ODAC voted 12 to 2 that the data in the NDA do not provide substantial evidence of effectiveness of L-MTP-PE in the treatment of patients with non-metastatic, resectable osteosarcoma receiving combination chemotherapy. The FDA will consider ODAC's recommendation when reviewing the NDA for L-MTP-PE. The Company anticipates a decision in late August 2007.
IDM Pharma is also seeking approval from the European Medicines Agency (EMEA) for the use of L-MTP-PE, or MEPACT as it is known in Europe. The Marketing Authorization Application (MAA) for L-MTP-PE was submitted to the EMEA on November 1, 2006 and accepted for review on November 27, 2006. The EMEA application is currently under review and the Company continues to work closely with the regulatory body to ensure they have the information needed to approve what would be the first new treatment option for osteosarcoma in nearly 20 years. L-MTP-PE was granted orphan drug status in Europe in 2004.
About 3% 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 tumors 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 tumor grows.
Osteosarcoma is an orphan disease with fewer than 1,000 new cases diagnosed in the U.S. each year. A similar incidence of the disease exists in Europe. According to the Children's Oncology Group, the survival of children with osteosarcoma has remained at 60-65% since the mid-1980s. The standard treatment for osteosarcoma is tumor resection with combination chemotherapy before and after surgery.
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