FDA Grants Multiple Myeloma Drug Priority Review

Ixazomib is the first investigational oral proteasome inhibitor for relapsed and/or refractory multiple myeloma.

The FDA on Wednesday granted priority review status to a new treatment option for multiple myeloma. Ixazomib, manufactured by Takeda Pharmaceutical Company, is the first investigational oral proteasome inhibitor therapy for relapsed and/or refractory multiple myeloma.

The drug is currently being evaluated for multiple myeloma, systemic light-chain amyloidosis (AL), and various other conditions. Ixazomib was previously granted orphan drug designation in the United States and Europe for multiple myeloma in 2011 and for AL amyloidosis in 2012.

The FDA granted ixazomib with Breakthrough Therapy designation for relapsed or refractory AL amyloidosis in 2014.

“We are encouraged that both the US and European regulatory bodies have determined that the ixazomib applications qualify for an expedited review, underscoring the importance of new treatment options for patients with relapsed/refractory multiple myeloma,” said Melody Brown, vice president of Regulatory Affairs at Takeda. “Our ixazomib program is designed to evaluate whether sustained therapy with an oral proteasome inhibitor improves the outcomes of patients living with multiple myeloma. There is a significant unmet medical need in multiple myeloma and we look forward to working with the regulatory bodies to bring ixazomib to patients.”

The priority review for ixazomib followed the results of the pivotal TOURMALINE-MM1 phase 3 trial. The international, randomized, double-blind, placebo controlled trial included 722 patients with relapsed and/or refractory multiple myeloma.

The study analyzed the efficacy of ixazomib plus lenalidomide and dexamethasone versus placebo plus lenalidomide and dexamethasone. Patients in the trial are still undergoing treatment to progression, with evaluations to come for long-term outcomes.