FDA Grants Breakthrough Therapy Status To Takeda Pharmaceutical Co. Ltd.'s Ixazomib

Published: Dec 08, 2014

FDA Grants Breakthrough Therapy Status to Takeda's Ixazomib

December 5, 2014

By Krystle Vermes, BioSpace.com Breaking News Staff

Takeda Pharmaceutical announced today that its oral proteasome inhibitor, Ixazomib, has received Breakthrough Therapy status from the U.S. Food and Drug Administration. With this status, the drug will be expedited for the sake of treating serious or life-threatening conditions.

Ixazomib, which is also known as MLN9708, is designed for the treatment of relapsed of refractory systemic light-chain amyloidosis. This is the first inhibitor and first investigational therapy for the condition to receive Breakthrough Therapy designation from the FDA. The development program for the drug has already progressed directly from a Phase 1 to a Phase 3 clinical trial.

"Patients with AL amyloidosis face a debilitating disease that can affect many of their organs and tissues,” said Raymond Comenzo, director of the Blood Bank and Stem Cell Processing Laboratory and professor at the Tufts University School of Medicine. "The Breakthrough Therapy designation for ixazomib is a major milestone in the development of new treatment options for patients battling this rare and aggressive disease. We are encouraged by positive preliminary data evaluating use of ixazomib in the treatment of patients with relapsed or refractory AL amyloidosis."

The data used to support the Breakthrough Therapy designation of Ixazomib will be presented at the American Society of Hematology annual meeting in San Francisco. This information includes the results of the Phase 1 and Phase 3 studies thus far.

"This Breakthrough Therapy designation is also an important recognition of the strength of our oncology development program and our commitment to extend proteasome inhibition to conditions that have not traditionally been the focus of research and development," said Michael Vasconcelles, head of the Oncology Therapeutic Area Unit at Takeda. "We would like to acknowledge and thank the patients, study investigators, and institutions whose participation in this program have made this milestone possible."

AL amyloidosis is a protein misfolding disorder that results in the deposition of amyloid in organs and tissues throughout the body. More than 3,000 cases of the disease are diagnosed in the U.S. each year. The condition typically impacts the heart, kidneys, liver, and spleen, currently, there are no approved treatments in the U.S. for the disease.

"Ixazomib is a breakthrough drug showing activity and organ improvement in most of these heavily pretreated AL amyloidosis patients," said Giampaolo Merlini, director of the Center for Research and Treatment of Systemic Amyloidosis at the University of Pavia.

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