The FDA ( Food and Drug Administration ) has 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.
Ixazomib 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 corticosteroid.
The safety and efficacy of Ixazomib 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 Ixazomib 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. ( Xagena )
Source: FDA, 2015