Active Ingredient History
PF-04449913 is a potent and selective inhibitor of the Hh signaling pathway through binding to the target, smoothened. PF-04449913 inhibits Hh signaling in vitro and has demonstrated significant antitumor activity in vivo. In the clinic, PF-04449913 is being evaluated both in hematological and solid malignancies, with a phase II trial currently underway in both fit and unfit patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). Treatment-related adverse-events were nausea, dizziness, somnolence, QT prolongation and pruritus. Based on pre-clinical assessments, CYP3A4 is believed to be primarily involved in the metabolism of PF-04449913 that is why PF-04449913 plasma exposures and peak concentrations were increased following concurrent administration of ketoconazole (CYP3A4 inhibitor). NCATS
Drug Pricing (per unit)
Note: This drug pricing data is preliminary, incomplete, and may contain errors.
Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
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Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
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Leukemia, Myeloid, Acute (approved 2018)
Fasciitis (Phase 1/Phase 2)
Glioblastoma (Phase 1/Phase 2)
Graft vs Host Disease (Phase 1/Phase 2)
Healthy Volunteers (Phase 1)
Hematologic Neoplasms (Phase 1)
Kidney Failure, Chronic (Phase 1)
Leukemia, Myeloid, Acute (Phase 3)
Leukemia, Myelomonocytic, Chronic (Phase 3)
Liver Diseases (Phase 1)
Myelodysplastic Syndromes (Phase 3)
Neoplasms (Phase 1)
Polycythemia Vera (Phase 2)
Sarcoma (Phase 3)
Trial | Phase | Start Date | Organizations | Indications |
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