FDA Approves Pomalidomide (Pomalyst) for Multiple Myeloma

February 11, 2013

The FDA has approved pomalidomide (Pomalyst) to treat patients with relapsed or refractory multiple myeloma who have received at least two prior therapies.

The US Food and Drug Administration (FDA) approved pomalidomide (Pomalyst) last week to treat patients with relapsed or refractory multiple myeloma who have received at least two prior therapies, including lenalidomide and bortezomib (Velcade). The new drug, a derivative of thalidomide, is an anti-angiogenic oral treatment that also acts as an immunomodulator.

“Pomalyst is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide, and is the second drug approved in the past year to treat multiple myeloma,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, in a press release.

In July 2012, FDA approved carfilzomib (Kyprolis)-a novel, highly selective epoxyketone proteasome inhibitor that is administered intravenously-to treat patients with multiple myeloma who have received at least two prior therapies, including treatment with bortezomib and an immunomodulatory therapy.

“Treatment for multiple myeloma is tailored to meet individual patients’ needs,” said Pazdur. “Today’s approval provides an additional treatment option for patients who have not responded to other drugs.”

The trial that led to the approval of pomalidomide was a phase II clinical trial of 221 patients (median age of 65 years) with relapsed or refractory multiple myeloma with measurable levels of M-protein. The primary trial endpoint was objective response rate. Patients were randomly assigned to receive pomalidomide alone (n = 109) at 4 mg (days 1–21 of a 28-day cycle), or pomalidomide with low-dose dexamethasone (40 mg), a corticosteroid, once a week (n = 113).

Trial results showed a 29.2% objective response rate in patients treated with pomalidomide plus low-dose dexamethasone vs 7.4% of patients treated with pomalidomide alone. A 7.4-month median duration of response was observed in the combination arm. Median progression-free survival was 3.8 months in the combination arm vs 2.5 months in the pomalidomide-alone arm (P = .007). Overall survival was 14.4 months with the combination treatment compared with 13.6 months with pomalidomide alone (P = .85).

Grade 3/4 adverse events seen with the combination include neutropenia (38%), anemia (21%), and thrombocytopenia (18%). Other common side effects include back pain (8%), fatigue and weakness, constipation, diarrhea, fever, pneumonia, and dyspnea.

The new treatment carries a boxed warning-pomalidomide can cause blood clots and should not be used in pregnant women as it can cause severe life-threatening birth defects.

Multiple myeloma, which primarily affects older adults, is a systemic malignancy of plasma cells. The disease typically arises within the bone marrow, secreting all or part of a monoclonal antibody. In 2012, approximately 21,700 patients were diagnosed with multiple myeloma in the United States, according to the National Cancer Institute, and it is estimated that 10,710 died from the disease.