On Feb. 8, the FDA approved pomalidomide (Pomalyst, Celgene Corporation) for patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression within 60 days of the last treatment, according to an agency press release.
“Treatment for multiple myeloma is tailored to meet individual patient’s needs, and today’s approval provides an additional treatment option for patients who have not responded to other drugs," according to Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.
A thalidomide analog, pomalidomide was approved under the FDA’s accelerated approval program and was granted orphan product designation. According to FDA and Celgene press releases, pomalidomide’s safety and effectiveness were evaluated in a Phase II, randomized, open-label trial of 221 patients with relapsed or refractory multiple myeloma. Patients were randomized to pomalidomide alone or pomalidomide with low-dose dexamethasone. In patients treated with pomalidomide alone, 7.4% achieved objective response rate (ORR). The median duration of response has not yet been reached in this arm. In patients treated with pomalidomide with low-dose dexamethasone, 29.2% achieved ORR; median duration of response was 7.4 months.
Approval is based on response rate, according to Celgene. Clinical benefit, such as improvement in symptoms or survival, has not been verified.
Pomalidomide carries a boxed warning stating that it is contraindicated in pregnancy because it causes severe birth defects or embryo-fetal death and that it can cause venous thromboembolism. Because of the embryo-fetal risk, pomalidomide is available only through the Pomalyst Risk Evaluation and Mitigation Strategy program. Common adverse reactions include fatigue and asthenia, neutropenia, anemia, constipation, nausea, diarrhea, dyspnea, upper respiratory tract infections, back pain and pyrexia.