The U.S. Food and Drug Administration approved Pomalyst (pomalidomide) to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs ( relapsed and refractory multiple myeloma ).
Pomalidomide (marketed as Pomalyst by Celgene), is a derivative of thalidomide that is anti-angiogenic and also acts as an immunomodulator.3-amino-thalidomide ( Pomalidomide ) was able to directly inhibit both the tumor cell and vascular compartments of myeloma cancers. This dual activity of pomalidomide makes it more efficacious than thalidomide in vitro and in vivo.
Pomalidomide directly inhibits angiogenesis and myeloma cell growth. This dual effect is central to it's activity in myeloma, rather than other pathways such as TNF alpha inhibition, since potent TNF alpha inhibitors including rolipram and pentoxifylline do not inhibit myeloma cell growth nor angiogenesis. Up regulation of Interferon gamma, IL-2 and IL-10 as well as down regulation of IL-6 have been reported for pomalidomide. These changes may contribute to pomalidomide's anti-angiogenic and anti-myeloma activities.
It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).
Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow.
“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, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “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.”
In July 2012, FDA approved Kyprolis (carfilzomib) to treat multiple myeloma.
Pomalyst’s safety and effectiveness was evaluated in a clinical trial of 221 patients with relapsed or refractory multiple myeloma. The trial was designed to measure 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 with low-dose dexamethasone, a corticosteroid.
Results showed 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 low-dose dexamethasone, 29.2 percent achieved ORR with a 7.4-month median duration of response.
Pomalyst, lenalidomide and thalidomide are marketed by Celgene, based in Summit, N.J. Kyprolis is marketed by South San Francisco, Calif.-based Onyx Pharmaceuticals.
Pomalyst was also granted orphan product designation because it is aimed at a rare disease/condition.
A Boxed Warning for Pomalyst (pomalidomide)
A Boxed Warning contained in the Pomalyst packaging alerts doctors and patients that the medication must not be given to pregnant mothers because of the serious risk of life-threatening birth defects (Pomalyst is an analogue of thalidomide), as well as increasing the possibility of blood clots.
Because of Pomalyst’s embryo-fetal risk, it is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the Pomalyst REMS Program by enrolling and complying with the REMS requirements. 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. Both lenalidomide and thalidomide have similar REMS.
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.Source :