The US Food and Drug Administration (FDA) has approved Pomalyst (pomalidomide) to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs. Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow.

Pomalyst is a pill that modulates 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, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).

“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. “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. Similar to Kyprolis, Pomalyst is being approved under the agency’s accelerated approval programme, 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 therapy was also granted orphan product designation because it is intended to treat a rare disease or condition.

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 per cent 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 per cent achieved ORR with a 7.4-month median duration of response.

The common side effects include a decrease in infection-fighting white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anaemia), constipation, diarrhoea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.

Pomalyst, lenalidomide and thalidomide are marketed by Celgene, based in Summit, New Jersey. Kyprolis is marketed by South San Francisco, California-based Onyx Pharmaceuticals.