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The Food and Drug Administration has granted breakthrough therapy designation to the investigational oral proteasome inhibitor ixazomib (MLN9708) for the treatment of patients with relapsed or refractory systemic light-chain (AL) amyloidosis.

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The drug is the first proteasome inhibitor and the first investigational therapy for AL amyloidosis to receive that the designation.


Approximately 1,200 to 3,200 new cases of AL amyloidosis are diagnosed in the U.S. each year, according to estimates from the Amyloidosis Foundation. And there are currently no approved treatments in the U.S. for the disease, notes a news release from the drug's manufacturer, Takeda Pharmaceutical Company Limited.


Ixazomib is currently being evaluated in combination with dexamethasone in the Phase III clinical trial TOURMALINE-AL1 for the treatment of patients with relapsed or refractory AL amyloidosis. The trial is the only Phase III trial for these patients currently ongoing, and the trial is enrolling patients globally.


Also given the Breakthrough Designation is JCAR015 for patients with relapsed or refractory B-cell acute lymphoblastic leukemia. The drug, made by Juno Therapeutics, is a chimeric antigen receptor product candidate, and Phase I clinical trials are currently ongoing, a news release notes.


The Breakthrough Therapy designation, enacted as part of the FDA's 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.