FDA Approves New Multiple Myeloma Rx

A newly approved treatment is the first cell-based gene therapy for multiple myeloma.

The US Food and Drug Administration (FDA) has approved Abecma (idecabtagene vicleucel) to treat adults who have multiple myeloma. This is the first treatment for multiple myeloma of its kind, the FDA noted.

“While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual’s age and the stage of the condition at the time of diagnosis," said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. "Today’s approval provides a new treatment option for patients who have this uncommon type of cancer.”

The FDA approved Abecma for use in adults with multiple myeloma that has not responded to at least four past types of treatment. This treatment is also approved for adults whose multiple myeloma came back after four or more past treatments.

Multiple myeloma is a rare blood cancer. It causes abnormal blood plasma cells to collect in the bone marrow and cause bone tumors.

Abecma is not a one-size-fits-all treatment for multiple myeloma. In fact, each time Abecma is used, it is customized for the patient. A medical professional collects the patient's T-cells (an important cell in the immune system). These cells are modified in a way that enables them to kill myeloma cells. The cells are then injected back into the patient.

The FDA based its approval of Abecma on a trial of 127 adults who had multiple myeloma that had returned after or had not responded to prior treatment. Around 72 percent of these patients saw a partial or full response after treatment with Abecma, according to the FDA. In total, 28 percent saw a complete response, meaning the multiple myeloma went away completely. And 65 percent of those who saw a complete response remained free of multiple myeloma for at least a year after treatment.

Abecma was linked to some potential side effects. In fact, this drug comes with a boxed warning — the most serious type of drug warning — about the risk of several potentially deadly conditions that can occur after receiving this treatment.

Common side effects of Abecma included cytokine release syndrome, a weakened immune system, pain and tiredness.

The FDA approved Abecma for Celgene Corporation.

Talk to your health care provider about the right treatment for your condition.