Takeda receives Breakthrough designation for ixazomib

Japanese drug major Takeda Pharmaceutical’s (TYO: 4502) has been granted Breakthrough Therapy designation from the US Food and Drug Administration for its oral proteasome inhibitor, ixazomib (MLN9708), for the treatment of relapsed or refractory systemic light-chain (AL) amyloidosis.

This is the first proteasome inhibitor and first investigational therapy for AL amyloidosis to receive Breakthrough Therapy designation. Compounds that receive Breakthrough Therapy status receive more intensive FDA guidance on an efficient drug development program. The current Phase III clinical trial, TOURMALINE-AL1, is evaluating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis. This is the only Phase III trial for relapsed or refractory AL amyloidosis and it is recruiting globally.

Michael Vasconcelles, head of the Oncology Therapeutic Area Unit at Takeda, said: "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."