Richard Pazdur, MD
The FDA announced the approval of pomalidomide (Pomalyst) for the treatment of multiple myeloma after patients have relapsed or become refractory to other cancer drugs.
Pomalidomide is an immunomodulatory agent as well as an inhibitor of angiogenesis, or the formation of new blood vessels in a tumor. The drug is able to halt the growth of blood vessels as well as stimulate the immune system. It is in the same family of drugs as thalidomide and lenalidomide (Revlimid), which are both approved to treat multiple myeloma as well.
The FDA noted that pomalidomide is intended for patients who have received at least two prior treatments for multiple myeloma, including lenalidomide and bortezomib (Velcade), and whose disease did not respond to treatment and progressed within 60 days of their last treatment.
The most recent treatment approved for multiple myeloma was carfilzomib (Kyprolis), which was approved by the FDA in July 2012. Both drugs were approved through the FDA’s accelerated approval program.
“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 statement. “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.”
The approval was based in large part on the results of a phase II trial that was presented at the 2012 Annual Meeting of the American Society of Clinical Oncology.
In that study, 221 patients were randomized 1:1 to receive either pomalidomide plus low-dose dexamethasone (n = 113) or pomalidomide alone (n = 108). In the pomalidomide combination arm of the study, patients received pomalidomide daily on days 1-21 at a dosage of 4 mg/d plus 40 mg/week of low-dose dexamethasone. The dose of pomalidomide was the same in both arms.
The study found that the objective response rate (ORR) in patients who receive pomalidomide alone was 7.4%, though the median duration of response had not yet been reached in these patients. In patients who received the combination, the ORR was 29.2% with a 7.4-month median duration of response.
Potential side effects associated with pomalidomide include neutropenia, thrombocytopenia, fatigue and weakness, anemia, constipation, diarrhea, upper respiratory tract infections, back pain, and fever.
The FDA has given the drug a Boxed Warning because the drug has the potential to cause severe life-threatening birth defects as well as blood clots. The Pomalyst Risk Evaluation and Mitigation Strategy (REMS) program has been established to determine which patients are able to receive the drug based on this warning.
Many hematologists and oncologists were hopeful that pomalidomide would be approved based on previous findings in clinical trials, including Sundar Jagannath, MD, director of the Multiple Myeloma Program at The Tisch Cancer Institute at The Mount Sinai Medical Center in New York, NY.
“This is a critical molecule that is important for patients who have currently failed all the existing treatment modalities,” Jagannath said.
Pomalyst is marketed by Summit, NJ-based Celgene.
Vij R, Richardson PGG, Jagannath S, et al. Pomalidomide (POM) with or without low-dose dexamethasone (LoDEX) in patients (pts) with relapsed/refractory multiple myeloma (RRMM): outcomes in pts refractory to lenalidomide (LEN) and/or bortezomib (BORT). J Clin Oncol. 2012;30(suppl; abstr 8016).