New Therapeutic for Treating Multiple Myeloma

Ixazomib is the first medication of its kind to be given to patients orally

The U.S. Food and Drug Administration (FDA) has approved ixazomib (Ninlaro) for treating certain patients with multiple myeloma.

The new anticancer therapeutic was approved for use in combination with lenalidomide (Revlimid) and dexamethasone. The three-drug treatment regimen is intended for patients with multiple myeloma that has progressed despite treatment with at least one prior therapy.

The ixazomib-containing combination of treatments delayed disease progression for patients in a randomized, double-blind phase III clinical trial.

Multiple myeloma is one of the most commonly diagnosed hematological malignancies, or blood cancers. The National Cancer Institute (NCI) estimates that there will be 26,850 new cases of multiple myeloma and 11,240 multiple myeloma?related deaths in the United States this year.

New therapeutics for multiple myeloma are urgently needed. Despite the development and FDA approval of new treatments for these patients ? including proteasome inhibitors like bortezomib (Velcade) and carfilzomib (Kyprolis), and immunomodulatory agents like lenalidomide (Revlimid) and pomalidomide (Pomalyst) ? which have improved patient outcomes in recent years, the overall five-year survival rate remains under 50 percent.

Ixazomib is the first oral proteasome inhibitor. As the name suggests, proteasome inhibitors block the function of a cellular complex called the proteasome. The function of the proteasome is to break down proteins in a cell, and this helps the cell control key processes such as cell division and survival. Preventing the natural breakdown of proteins can be highly toxic to myeloma cells, causing them to die.

According to the FDA announcement, the approval of ixazomib was based on results from a phase III clinical trial, which showed that adding ixazomib to lenalidomide and dexamethasone increased the time by almost six months before disease progressed.

Ixazomib was the third new therapeutic approved by the FDA in 2015 for treating multiple myeloma, after panobinostat (Farydak) and daratumumab (Darzalex). After ixazomib’s approval, the FDA went on to approve elotuzumab, adding to the growing roster of options to treat multiple myeloma.

The FDA approval was rendered on Nov. 20, 2015.