On January 8 the U.S. FDA approved Pomalyst (pomalidomide) for the treatment of patients with multiple myeloma whose disease has progressed despite treatment with other anti-cancer drugs. Multiple myeloma is a blood cancer that primarily affects the aging population and is induced from plasma cells in the bone marrow. The National Cancer Institute estimates that approximately 21,700 Americans are diagnosed with multiple myeloma each year and 10,710 die from the disease. Pomalyst is a pill that regulates the body’s immune system, destroying cancer cells and inhibiting their growth. It is appropriate for patients who have received at least two prior therapeutic agents, including lenalidomide and bortezomib, who have not responded to treatment (failed to work) and who have progressed within 60 days of the last treatment (relapsed and refractory). Pomalyst is the third drug in the immunomodulator class, after lenalidomide and thalidomide. Treatment for multiple myeloma needs to be “tailored” to meet the needs of individual patients, and the approval of Pomalyst provides a new option for patients who have failed to respond to other medications. In July 2012, the FDA previously approved Kyprolis (carfilzomib) for the treatment of multiple myeloma. Similar to Kyprolis, Pomalyst was approved through the FDA’s accelerated approval process and received rare drug treatment. The safety and efficacy of Pomalyst was evaluated in a clinical trial involving 221 patients with relapsed or refractory multiple myeloma. The purpose of the trial was to look at the number of patients whose cancer completely or partially disappeared after treatment (objective response rate, or ORR). Patients were randomly assigned to receive Pomalyst alone or Pomalyst plus the low-dose corticosteroid dexamethasone (dexamethasone). The results showed that 7.4% of patients treated with Pomalyst alone achieved an ORR, and the median duration of response in these patients was inconclusive (still responding at the time of enumeration). 29.2% of patients treated with Pomalyst plus low-dose dexamethasone showed an ORR with a median duration of response of 7.4 months. Pomalyst’s label carries a framed warning to patients and healthcare professionals that the drug should not be given to pregnant women because it may cause life-threatening serious birth defects and that the drug may cause blood clots. Because of the risk to the fetus, Pomalyst is dispensed through the Risk Evaluation and Mitigation Program (REMS): signed by the physician and patient as required by the REMS. In particular, pregnancy tests and contraception are required for female patients who are not pregnant and may conceive, and male patients must use contraception as required. Pharmacies need to be certified in accordance with REMS and the drug can only be prescribed to patients who meet the requirements. Common side effects include decreased infection-fighting white blood cells (neutrophils), fatigue and weakness, decreased red blood cell count (anemia), constipation, diarrhea, decreased thrombocytopenia (thrombocytopenia), upper respiratory tract infections, back pain, and fever. Pomalyst, lenalidomide and thalidomide are marketed by Celgene Corporation and Kyprolis is marketed by Onyx Pharmaceuticals, Inc.