Silver Spring, MD (Press Release) – Today the U.S. Food and Drug Admin­istra­tion granted approval for Ninlaro (ixazomib) in com­bi­na­tion with two other ther­a­pies to treat people with multiple myeloma who have received at least one prior ther­apy.

Multiple myeloma is a form of blood cancer that occurs in in­fec­tion-fighting plasma cells (a type of white blood cell) found in the bone marrow. These can­cer­ous cells multiply, produce an ab­nor­mal protein and push out other healthy blood cells from the bone marrow. The disease may result in a weakened immune system and cause other bone or kidney problems. The National Cancer Institute esti­mates there will be 26,850 new cases of multiple myeloma and 11,240 related deaths in the United States this year.

“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, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “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.” The FDA approved Farydak (panobinostat)in February and Darzalex (dara­tu­mu­mab) earlier this month.

Ninlaro is a type of cancer drug called a pro­te­a­some inhibitor and works by blocking enzymes from multiple myeloma cells, hindering their ability to grow and survive. Ninlaro is the first oral pro­te­a­some inhibitor and is approved in com­bi­na­tion with another FDA-approved treat­ment for multiple myeloma called Revlimid (lena­lido­mide) and dexa­meth­a­sone (a type of corticosteroid).

The safety and efficacy of Ninlaro were dem­onstrated in an inter­na­tional, ran­domized, double-blind clin­i­cal trial of 722 patients whose multiple myeloma came back after, or did not respond to, pre­vi­ous treat­ment. Study par­tic­i­pants received either Ninlaro in com­bi­na­tion with lena­lido­mide and dexa­meth­a­sone or placebo plus lena­lido­mide and dexa­meth­a­sone. Those taking Ninlaro lived longer without their disease worsening (average 20.6 months) compared to par­tic­i­pants taking the other regi­men (14.7 months).

The most common side effects of Ninlaro are diarrhea, con­sti­pa­tion, low blood platelet count (thrombocytopenia), periph­eral neu­rop­athy (numbness and pain from nerve damage, usually in the hands and feet), nausea, periph­eral edema (fluid under the skin causing swelling), vomiting and back pain.

The FDA granted priority review and orphan drug desig­na­tions for Ninlaro. Priority review status is granted to appli­ca­tions for drugs that, if approved, would be a sig­nif­i­cant im­prove­ment in safety or effectiveness in the treat­ment of a serious con­di­tion. Orphan drug desig­na­tion provides incentives such as tax credits, user fee waivers, and eligibility for orphan drug exclusivity to assist and encourage the devel­op­ment of drugs for rare diseases.

Ninlaro is marketed by Takeda Pharma­ceu­ticals based in Osaka, Japan. Farydak is marketed by East Hanover, New Jersey-based Novartis Pharma­ceu­ticals. Darzalex is marketed by Janssen Biotech of Horsham, Pennsylvania. Revlimid is marketed by Celgene Corpo­ra­tion, based in Summit, New Jersey.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological prod­ucts for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supple­ments, prod­ucts that give off electronic radiation, and for regulating tobacco prod­ucts.

Source: The Food and Drug Admin­istra­tion.