Takeda’s Ninlaro Gets Approval from US FDA for Multiple Myeloma

On 20th Nov 2015 the U.S. FDA provided acceptance for download (ixazomib) in combo with two other therapies to treat patients with multiple myeloma who have obtained minimum one prior treatment. Multiple myeloma is a type of blood cancer that develops in infection-fighting plasma cells (a form of white blood cell) identified in the bone marrow. These cancer cells grow, generate an abnormal protein and push out other healthy blood cells from the bone marrow. The condition may outcome in a weakened immune system and lead to other bone or kidney issues. The National Cancer Institute reports there will be more than 26000 new cases of multiple myeloma and 11,240 associated deaths in the United States in 2015.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research stated

As we understand more about the actual biology of multiple myeloma, we are inspired to see the growth of new methods to deal with this condition. Today’s acceptance is the 3rd drug for multiple myeloma accepted this year and offers sufferers with a new oral therapy that decreases disease development when other treatment has failed.” The FDA accepted 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 confirmed in an global, randomized, double-blind clinical study of 722 sufferers whose multiple myeloma came back following, or failed to respond to, earlier treatment. Study individuals obtained either Ninlaro in combo with lenalidomide and dexamethasone or placebo plus lenalidomide and dexamethasone. Those obtaining Ninlaro lived more time without their condition worsening (average 20.6 months) in comparison to individuals taking the other regimen (14.7 months).

The very common side effects of Ninlaro are constipation, diarrhea, thrombocytopenia, peripheral neuropathy, nausea, peripheral edema , vomiting and back ache.

The US FDA provided priority review and orphan drug status for Ninlaro. Priority review designation is provided to applications for drugs that, if accepted, would be a considerable improvement in safety or effectiveness in the therapy of a serious problem. Orphan drug designation offers benefits such as tax credits, user fee waivers, and to be eligible for orphan drug exclusivity to assist and motivate the development of medicines for rare conditions. Ninlaro is sold by Takeda Pharmaceuticals.