Posted on March 13th, 2013 by
The U.S. Food and Drug Administration (FDA) has approved Pomalyst® (pomalidomide) for the treatment of patients with multiple myeloma whose disease progressed after being treated with other cancer drugs.
Multiple myeloma is a cancer of plasma cells, which are a special type of white blood cell that are part of the body’s immune system. Patients with multiple myeloma have increased numbers of abnormal plasma cells that may produce increased quantities of dysfunctional antibodies detectable in the blood and/or urine. Patients with multiple myeloma who have become refractory—or resistant—to the drugs Revlimid® (lenalidomide) and Velcade® (bortezomib) have limited treatment options. There is no standard treatment for these patients and they typically have a poor prognosis, with a median overall survival of 9 months.
Pomalyst is an immunomodulatory drug that has shown activity in multiple myeloma patients who have become refractory to Velcade®, Thalomid® (thalidomide), and Revlimid®. It works by directly inhibiting angiogenesis and myeloma cell growth. Pomalyst is delivered as an oral pill and it causes the body’s immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).
The approval was based on the results of a study that included 221 patients with relapsed or refractory multiple myeloma. Patients were randomly assigned to receive Pomalyst alone or Pomalyst plus low-dose dexamethasone (LoDEX). The study was designed to determine the number of patients whose cancer completely or partially disappeared after treatment (objective response rate, or ORR).
The results indicated that 7.4 percent of patients treated with Pomalyst alone achieved ORR. The median duration of response has not yet been reached in these patients. In patients treated with Pomalyst plus LoDEX, 29.2 percent achieved ORR with a 7.4-month median duration of response.
Common side effects include a decrease in infection-fighting white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anemia), constipation, diarrhea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.
Pomalyst is being approved under the agency’s accelerated approval program, which provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug’s clinical benefit and safe use. The drug was also granted orphan product designation because it is intended to treat a rare disease.
Pomalyst carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can cause severe life-threatening birth defects, and that the drug can cause blood clots.
Because of the risk to fetuses, Pomalyst is only available through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the Pomalyst REMS Program. Patients must sign a Patient-Physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS Program, must only dispense to patients who are authorized to receive the drug and must comply with REMS requirements.
FDA approves Pomalyst for advanced multiple myeloma. [FDA News Release]. U.S. Food and Drug Administration website. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm338895.htm
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