Approval Announcement
November 20, 2015 -- 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.
Overview of Multiple Myeloma
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.
FDA Official Statement on the Approval
“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.”
Notably, the FDA approved Farydak (panobinostat) in February and Darzalex (daratumumab) earlier this same month prior to Ninlaro’s approval.
Mechanism of Action and Regimen Details
Ninlaro is a type of cancer drug classified as a proteasome inhibitor. It works by blocking enzymes from multiple myeloma cells, hindering their ability to grow and survive. A landmark feature of Ninlaro is that it is the first oral proteasome inhibitor approved for this indication.
Ninlaro is approved for use in combination with two other FDA-approved agents for multiple myeloma: Revlimid (lenalidomide), an immunomodulatory drug, and dexamethasone, a corticosteroid.
Clinical Trial Efficacy and Safety Data
Efficacy Support
The safety and efficacy of Ninlaro were demonstrated in an international, randomized, double-blind clinical trial involving 722 patients with relapsed or refractory multiple myeloma (disease that came back after, or did not respond to, previous treatment). Study participants were randomized to receive either Ninlaro plus lenalidomide and dexamethasone, or placebo plus lenalidomide and dexamethasone.
Patients treated with the Ninlaro combination regimen had a significantly longer progression-free survival, with a median of 20.6 months without disease worsening, compared to 14.7 months in the placebo combination group.
Most Common Adverse Reactions
The most common side effects associated with Ninlaro include 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.
FDA Designations Granted to Ninlaro
The FDA granted both priority review and orphan drug designations for Ninlaro. Priority review is assigned to drugs that, if approved, would offer a significant improvement in safety or effectiveness for treating serious conditions. Orphan drug designation provides targeted incentives including tax credits, user fee waivers, and eligibility for orphan drug exclusivity, to support and promote the development of therapies for rare diseases.
Marketing Authorization Holders
Ninlaro is marketed by Takeda Pharmaceuticals, headquartered in Osaka, Japan. Additional related agents are marketed by respective companies: Farydak by Novartis Pharmaceuticals (East Hanover, New Jersey), Darzalex by Janssen Biotech (Horsham, Pennsylvania), and Revlimid by Celgene Corporation (Summit, New Jersey).
