Takeda amyloidosis therapy granted breakthrough status by US FDA

Takeda’s investigational, oral proteasome inhibitor, ixazomib (MLN9708), for the treatment of relapsed or refractory systemic light-chain (AL) amyloidosis, has received Breakthrough Therapy designation from the US Food and Drug Administration (FDA).

The drug candidate moved straight from phase I to phase III clinical trial, TOURMALINE-AL1, which is the only phase III trial for relapsed or refractory AL amyloidosis and is recruiting globally. If successful, it will meet a significant unmet medical need.

“Patients with AL amyloidosis face a debilitating disease that can affect many of their organs and tissues. 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 Dr Raymond Comenzo, director, Blood Bank and Stem Cell Processing Laboratory and professor at Tufts University School of Medicine.

The therapy showed “activity and organ improvement” in most of the trial participants, according to Prof Giampaolo Merlini, director, Center for Research and Treatment of Systemic Amyloidosis, University of Pavia, Italy.

AL amyloidosis is a rare and aggressive protein misfolding disorder. Fewer than 3,000 cases are diagnosed in the US each year. It is characterised by the deposition of amyloid in bodily organs and tissues. It frequently affects the heart, kidneys, liver, spleen, nervous system and gastrointestinal tract.

The head of Takeda’s Oncology Therapeutic Area Unit, Michael Vasconcelles said that the Breakthrough Therapy designation was also a recognition of the strength of the company’s oncology development programme and its commitment to extend proteasome inhibition to conditions that have not traditionally been the focus of research and development.

Takeda had a setback earlier this year in its Millennium unit portfolio, with its most advanced prostate cancer candidate, orteronel, being terminated after it failed to show any significant survival improvements. But the company has great hopes for Ixazomib in multiple myeloma (MM), which it plans will succeed blockbuster Velcade. It also has alisertib (MLN8237) in phase III trials for peripheral T-cell lymphoma.

Ixazomib is also being studied in systemic light-chain (AL) amyloidosis. It was granted orphan drug designation in MM in the US and Europe in 2011, and for AL amyloidosis in the US and Europe in 2012.


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