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Therapeutic efficacy of Ixazomib

Ixazomib (Ninlaro) is an antineoplastic agent classified as a proteasome inhibitor. Its mechanism of action involves blocking relevant enzymes in multiple myeloma cells, thereby inhibiting the growth and survival capacity of tumor cells. As the first oral proteasome inhibitor, ixazomib is currently approved for use in combination with two other drugs approved by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma. These two drugs are lenalidomide (brand name: Revlimid) and dexamethasone (a glucocorticoid).

The safety and efficacy of ixazomib have been verified through an international, multicenter, randomized, double-blind clinical trial involving 722 participants. All enrolled patients had relapsed or refractory multiple myeloma following prior treatment. The subjects were randomly divided into two groups: one group received ixazomib in combination with lenalidomide and dexamethasone, while the other group received a placebo in combination with lenalidomide and dexamethasone. The results showed that patients in the ixazomib treatment group achieved a longer progression-free survival (PFS), with a median PFS of 20.6 months, compared with only 14.7 months in the control group.

The most common adverse reactions of ixazomib include diarrhea, constipation, thrombocytopenia, peripheral neuropathy (numbness and pain symptoms caused by nerve damage, most commonly affecting the hands and feet), nausea, peripheral edema (swelling caused by subcutaneous fluid accumulation), vomiting, and back pain.

The FDA has granted ixazomib priority review designation and orphan drug designation. Priority review designation is given to drugs that, if approved, are expected to provide a significant improvement in safety or effectiveness in the treatment of serious diseases. Orphan drug designation offers a series of incentives, including tax credits, application fee waivers, and orphan drug exclusivity rights, to support and encourage the research and development of drugs for rare diseases.

from FDA,2024.07