FDA Approves New Treatment for Refractory Multiple Myeloma

Ixazomib is an oral proteasome inhibitor used in combination with lenalidomide and dexamethasone.

The FDA today granted approval to a new treatment option for patients with multiple myeloma.

Ixazomib (Ninlaro) is an oral proteasome inhibitor used in combination with lenalidomide and dexamethasone for the treatment of multiple myeloma following progression on at least 1 prior therapy.

The efficacy of the drug was shown in the 722-patient phase 3 TOURMALINE-MM1 trial comparing treatment with ixazomib plus lenalidomide and dexamethasone versus 14.7 months with lenalidomide and dexamethasone alone.

Patients were administered 25 mg of oral lenalidomide on days 1-21 and 40 mg of oral dexamethasone on days 1, 8, 15, and 22 in combination with ixazomib at 4 mg on days 1, 8, and 15, or placebo. Patients were treated until disease progression was observed.

Patients enrolled in the trial received between 1 and 3 prior therapies with an ECOG PS of 0, 1, or 2. The results showed a median progression-free survival (PFS) of 20.6 months with ixazomib plus lenalidomide and dexamethasone compared with 14.7 months with lenalidomide and dexamethasone alone.

The addition of ixazomib did not cause a substantial increase in adverse events. Ixazomib was previously granted priority review by the FDA.

“As we learn more about the underlying biology of multiple myeloma, we are encouraged to see the development of new ways to treat this disease,” said Richard Pazdur, MD, director of the FDA Office of Hematology and Oncology Products. “Today’s approval is the third drug for multiple myeloma approved this year and provides patients with a new oral treatment that slows disease progression when other therapy has failed.”