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The compassionate access program provides L-MTP-PE treatment to patientswho have either failed or cannot tolerate treatment with existing therapiescompared to the Phase 3 pivotal study, which evaluated newly diagnosedpatients.
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"The results that we have seen to date from the Phase 3 clinical trial inpatients with non-metastatic osteosarcoma are being supported in patients withlung metastases as the L-MTP-PE compassionate access experience continues tobe encouraging," said Peter Anderson, M.D., Ph.D., principal investigator andprofessor of pediatrics, Children's Cancer Hospital at The University of TexasM. D. Anderson Cancer Center in Houston. "With a formalized protocol now inplace, we can address the increasing number of requests for compassionateaccess to L-MTP-PE and meet the unmet needs of certain patients by providingthis much needed treatment option."
Study design and findings
Twenty-nine high-risk osteosarcoma patients (ages 10 - 21) were enrolledand 27 have been treated with L-MTP-PE in the compassionate access program todate (May 8, 2008). Patients in the program had documented diagnosis of highgrade osteosarcoma with relapsed or recurrent disease (locally or metastatic)with resectable or not completely resectable disease, or who are unable tocomplete recommended chemotherapy due to toxicity.
L-MTP-PE (2 mg/m2 IV over 1 hour) was administered twice a week for 12weeks followed by once a week for 24 weeks. In addition, some patients in theprogram were also treated with other agents including aerosol recombinantgranulocyte monocyte colony stimulating factor (GM-CSF) an immune stimulatingagent (n=20), ifosfamide (n=4), and/or gemcitabine (n=2).
Treatment with L-MTP-PE combined with other agents including aerosol GM-CSF was generally well tolerated. Patients treated with chemotherapy had nounexpected toxicities and toxicity from L-MTP-PE infusions was minimal. Therewere no reports of grade 3 or 4 drug-related toxicities with the exception offever grade 3 and flu-like symptoms with the first dose. This was preventedwith ibuprofen and acetaminophen after subsequent doses. One patient developedpleural and pericardial effusion that was possibly L-MTP-PE and/or GM-CSFrelated and the patient was removed from the study.
In March, the Company announced that it had formalized a clinical protocolwith the FDA, which provides L-MTP-PE to eligible, high-risk osteosarcomapatients through a compassionate access study. The compassionate access studyis being conducted initially at M. D. Anderson and Memorial-Sloan KetteringCancer Center in New York.
"Patients are our first priority and we are committed to providing L-MTP-PE to those who desperately need treatment," said Timothy P. Walbert,president and chief executive officer, IDM Pharma. "The potential survivaland quality of life benefits for patients treated with L-MTP-PE continue to besupported through physician experience and we remain committed to advancing L-MTP-PE through the European and U.S. regulatory processes to bring thisimportant treatment to market."
Update on L-MTP-PE Regulatory Status
In January 2008 the Company announced that following presentation of dataat an oral explanation hearing before the Committee for Medicinal Products forHuman Use (CHMP) of the European Medicines Agency, the CHMP determined in anon-binding opinion that L-MTP-PE suggested a possib
