The US Food and Drug Administration has approved Takeda’s Ninlaro as the first oral proteasome inhibitor for the treatment of the incurable and rare blood cancer multiple myeloma.

Ninlaro (ixazomib) has been cleared for use in combination with Celgene’s Revlimid (lenalidomide) and dexamethasone to treat patients who have received at least one prior therapy, after clinical data showed that it offered a benefit in progression-free survival.

Patients taking the once-weekly regimen lived for an average of 20.6 months without their disease worsening compared to 14.7 months in the group receiving a placebo plus Revlimid and dexamethasone, with a tolerable safety profile.

The drug, which was awarded a priority review by the regulator and also carried Orphan drug status, works by blocking enzymes from multiple myeloma cells, hindering their ability to grow and survive. 

While there has been significant progress in treatment of the disease in recent years, there remains a significant unmet need. Ninlaro’s approval provides patients with “a new oral treatment that slows disease progression when other therapy has failed,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. 

“The introduction of Ninlaro marks an important step forward, as its efficacy and safety profile - coupled with its completely oral administration - potentially can reduce some logistical burdens, and help enable patients to reap the full benefits of this sustainable therapy,” noted Christophe Bianchi, president of Takeda Oncology.

The drug’s green light also marks the third for multiple myeloma in the US this year, following FDA clearance of Novartis’ Farydak (panobinostat) in February and Janssen Biotech’s Darzalex (daratumumab) earlier this month.

The National Cancer Institute estimates there will be 26,850 new cases of multiple myeloma and 11,240 related deaths in the US this year.