Three New Therapies Approved for Multiple Myeloma
January 6, 2016, by NCI Staff
The Food and Drug Administration (FDA) has approved three new drugs for the treatment of multiple myeloma that has returned after prior therapy.
On November 16, the FDA approved daratumumab (Darzalex®) for patients who have previously received at least three prior treatments. On November 20, the agency approved ixazomib (Ninlaro®) to treat patients with relapsed multiple myeloma who have received at least one prior treatment, and on November 30 it approved elotuzumab (Empliciti®) for patients who have received one to three prior therapies.
“Relapse is almost universal with myeloma,” explained Mark Roschewski, M.D., of the Lymphoid Malignancies Branch in NCI’s Center for Cancer Research. “Some patients enjoy long durations of remission after their first treatment, but very few patients can be cured today.”
The approval of ixazomib, a proteasome inhibitor, was based on the results of a large randomized clinical trial of 722 patients in which patients treated with ixazomib in combination with lenalidomide and dexamethasone had longer median progression-free survival than those who received lenalidomide and dexamethasone alone: 20.6 months versus 14.7 months.
“There are already two proteasome inhibitors [bortezomib and carfilzomib] that have been approved for the treatment of multiple myeloma,” said Dr. Roschewski. “But this is the first one that is available orally. So that’s a very important improvement.”
Daratumumab’s approval was based on two single-arm trials. In the first trial, 29 percent of patients experienced a complete or partial reduction in their tumor burden that lasted for a median of 7.4 months. In the second trial, 36 percent of patients had a complete or partial reduction in tumor burden.
The approval of elotuzumab was based on the results of a randomized clinical trial of 646 participants whose myeloma did not respond to or had relapsed after previous treatment. Patients treated with elotuzumab in combination with lenalidomide and dexamethasone had longer median progression-free survival than patients who received only lenalidomide and dexamethasone: 19.4 months versus 14.9 months.
“Elotuzumab by itself doesn’t have single-agent activity,” noted Dr. Roschewski. “But if you add it to other agents, elotuzumab extends progression-free survival.”
The side effects that occurred most often in patients taking the drugs included fatigue, diarrhea, peripheral neuropathy, and fever. Daratumumab may also cause a decrease in white blood cells.
Elotuzumab and daratumumab, both monoclonal antibodies, target proteins on myeloma cells that have yet to be part of other FDA-approved therapies for this cancer type, explained Dr. Roschewski.
“The most exciting of the three drugs is daratumumab, because it showed single-agent activity,” Dr. Roschewski said. “It is not, by itself, going to cure multiple myeloma, and the duration of remission is relatively short, but it certainly provides some meaningful benefit, particularly if a patient doesn’t have any other treatment options.
“Any time you can get three new medicines in your armamentarium, that opens up many new research opportunities that now need to be explored,” Dr. Roschewski said.