FDA grants breakthrough status to investigational, oral proteasome inhibitor for AL amyloidosis

Investigational, oral proteasome inhibitor, ixazomib (MLN9708, Takeda) has FDA has granted breakthrough therapy status 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.

Read more on the breakthrough therapy designation

“There are currently no approved treatments for patients in the United States or globally with relapsed or refractory systemic light-chain [AL] amyloidosis, and so 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,” said Michael Vasconcelles, MD, head, Oncology Therapeutic Area Unit, Takeda

The development program for ixazomib in this indication progressed directly from a phase 1 to a phase 3 clinical trial, TOURMALINE-AL1, which is currently evaluating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis. This is the only phase 3 trial for relapsed or refractory AL amyloidosis and it is recruiting globally.

The data used to support this designation will be presented at the American Society of Hematology (ASH) annual meeting to be held December 6-9, 2014 in San Francisco.

AL amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the United States every year. It is characterized by the deposition of amyloid in bodily organs and tissues. While AL amyloidosis can affect different organs in different people, it frequently affects the heart, kidneys, liver, spleen, nervous system, and gastrointestinal tract.