On 20 November, 2015 the US Food and Drug Administration (FDA) granted approval for ixazomib (Ninlaro) in combination with two other therapies (lenalidomide and dexamethasone) to treat patients with multiple myeloma who have received at least one prior therapy.
“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 Dr Richard Pazdur, 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 panobinostat (Farydak) in February and daratumumab (Darzalex) earlier this month.
Ninlaro is 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 lenalidomide (Revlimid) and dexamethasone.
The safety and efficacy of Ninlaro were demonstrated in an international, randomised, 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 obtained progression-free survival of average 20.6 months compared to participants taking the other regimen (14.7 months).
The most common side effects of Ninlaro are diarrhoea, constipation, thrombocytopenia, peripheral neuropathy, nausea, peripheral oedema, 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.