Celgene’s Multiple Myeloma Orphan Drug Gets FDA Approval

The US FDA has granted approval to Celgene’s Pomalyst (pomalidomide), a treatment for multiple myeloma, a form of blood cancer. This approval comes under the FDA’s accelerated approval program and includes orphan product designation due to its focus on treating a rare disease. Approximately 21,700 Americans are diagnosed with multiple myeloma each year, highlighting the importance of this new treatment option.

Pomalyst is designed for patients whose disease has progressed after being treated with at least two prior therapies, including lenalidomide and bortezomib, and who have not responded to these treatments. It works by modulating the body’s immune system to destroy cancerous cells and inhibit their growth.

Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, emphasized the significance of this approval: “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. Today’s approval provides an additional treatment option for patients who have not responded to other drugs.”

This development represents a critical advancement in the treatment of multiple myeloma, offering hope to patients who have exhausted other treatment options.