The U.S. FDA Grants Priority Review Status to Takeda’s Ixazomib for the Treatment of Relapsed/Refrac

On September 9, 2015, Cambridge, Massachusetts, USA, and Osaka, Japan, announced that the U.S. Food and Drug Administration (FDA) had granted priority review status to the New Drug Application (NDA) for ixazomib. As the first investigational oral proteasome inhibitor, ixazomib is intended for the treatment of patients with relapsed and/or refractory multiple myeloma.

Melody Brown, Vice President of Regulatory Affairs at Takeda Pharmaceutical Company Limited, stated: "We are greatly encouraged that both U.S. and European regulatory authorities have determined that ixazomib’s NDA meets the criteria for accelerated review, which underscores the importance of providing new treatment options for patients with relapsed/refractory multiple myeloma. Our ixazomib research program is designed to evaluate whether continuous administration of this oral proteasome inhibitor can improve outcomes for patients with multiple myeloma. Given the significant unmet medical needs that still exist in the field of multiple myeloma, we look forward to collaborating with regulatory authorities to bring ixazomib to patients as soon as possible."

The FDA may grant priority review status to NDAs for drugs intended to treat serious diseases if approval of such drugs would provide a significant improvement in safety or effectiveness compared to existing therapies. This status includes an accelerated review process. Recently, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has also granted accelerated assessment status to ixazomib.

The NDA for ixazomib is primarily based on the results of the first pre-specified interim analysis of the pivotal Phase III clinical trial TOURMALINE-MM1. This was an international, multicenter, randomized, double-blind, placebo-controlled clinical trial involving 722 patients, designed to demonstrate the superiority of the ixazomib-lenalidomide-dexamethasone combination regimen over the placebo-lenalidomide-dexamethasone combination regimen in the treatment of adult patients with relapsed and/or refractory multiple myeloma. Patients in this trial are still receiving treatment until disease progression, and their long-term outcomes will be assessed in subsequent analyses.

Ixazomib is an investigational oral proteasome inhibitor currently under study for the treatment of multiple myeloma, light chain (AL) amyloidosis, and other hematologic malignancies. In 2011, the drug was granted orphan drug designation for multiple myeloma by both U.S. and European regulatory authorities; in 2012, it was granted orphan drug designation for AL amyloidosis in both regions simultaneously; and in 2014, the U.S. FDA granted it breakthrough therapy designation for the treatment of relapsed/refractory AL amyloidosis. In addition, ixazomib is the first oral proteasome inhibitor to enter Phase III clinical trials.