Celgene’s multiple myeloma orphan drug gets FDA approval

Hannah Blake

pharmaphorum

Celgene’s multiple myeloma treatment has been approved by the US FDA. Pomalyst has been approved to treat patients with this form of blood cancer whose disease progressed after being treated with other cancer drugs.

Pomalyst (pomalidomide) was approved under the FDA’s accelerated approval program and it was also granted orphan product designation because it is intended to treat a rare disease or condition. Approximately 21,700 Americans are diagnosed with multiple myeloma a year, according to the National Cancer Institute.

“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. 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.”

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research.

Pomalyst is a pill that modulates the body’s immune system to destroy cancerous cells and inhibit their growth. According to the FDA, 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).