FDA approves Takeda's Ninlaro, the first and only oral proteasome inhibitor to treat multiple myeloma

Ninlaro provides a new option for patients living with multiple myeloma who have received at least one prior therapy.

Takeda submitted a New Drug Application for Ninlaro to the FDA in July 2015, and in September Ninlaro was granted Priority Review status with a PDUFA date of 10 March 2016, reflecting the profound and continuing unmet need for new treatments for multiple myeloma, a devastating, relapsing and incurable rare cancer.

“With the approval of Ninlaro, we can now offer patients a once-weekly oral proteasome inhibitor as part of a highly active triplet therapy,” said Paul Richardson, Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center Institute Physician at Dana-Farber Cancer Institute, and investigator for TOURMALINE-MM1, the pivotal Phase III trial on which the FDA approval is based. “We, as investigators of the TOURMALINE-MM1 trial, felt it was vital to conduct a comprehensive ‘real world’ evaluation of this combination that included some of the most common patient types in the relapsed/refractory multiple myeloma setting, such as older patients, patients with moderate renal impairment, light chain disease, and high risk cytogenetics. Further, we treated patients until disease progression to determine the sustainability of Ninlaro in treating their relapsed/refractory disease. The TOURMALINE-MM1 data demonstrate convincingly that oral Ninlaro-based triplet treatment is effective at extending progression-free survival, over and above the clinical benefit seen with lenalidomide and dexamethasone, with a tolerable safety profile.”

“We introduced the first proteasome inhibitor for multiple myeloma, VELCADE, into clinical research approximately 20 years ago. Since that time, we’ve significantly advanced scientific understanding of this rare cancer, culminating in the introduction of Ninlaro,” said Andy Plump, Takeda Chief Medical and Scientific Officer. “Ninlaro is an entirely new molecule that offers the efficacy of this proteasome inhibitor in a convenient once-weekly pill with a tolerable safety profile. Takeda is delighted to bring this significant innovation to multiple myeloma patients today, and we continue to examine the potential of Ninlaro through a robust clinical development program.”