Pomalidomide for patients with multiple myeloma.

Multiple myeloma is a malignancy of plasma cells in the bone marrow. Currently, multiple myeloma is not considered curable, but it is treatable with different strategies that can combine chemotherapy with autologous hematopoietic stem cell transplantation. Pomalidomide is an orally active thalidomide analogue that has a pleiotropic mechanism of action involving oncolytic, antiangiogenic, immunomodulatory and anti-inflammatory activities. Pomalidomide is extensively metabolized, mainly by the cytochrome P450 3A4 and 1A2 pathways. The safety and efficacy of pomalidomide combined with dexamethasone has been demonstrated in a phase III trial for the treatment of multiple myeloma patients, relapsed/resistant to bortezomib and lenalidomide. Adverse events that were mainly related to myelosuppression, were manageable. Pomalidomide has orphan drug status both in the U.S. and Europe for multiple myeloma. It was approved by the U.S. Food and Drug Administration as Pomalyst® for the treatment of multiple myeloma last February, and recently approved in Europe in August.