The US Food and Drug Administration has assigned Takeda’s ixazomib (MLN9708) ‘breakthrough’ status for the treatment of relapsed or refractory systemic light-chain (AL) amyloidosis. 

The move indicates that the regulator believes the drug may offer a substantial improvement to patients after a promising performance in early-stage trials, and could fulfil a significant unmet medical need given that there are no treatments approved for AL amyloidosis anywhere in the world.

The disease is a rare and aggressive protein mis-folding disorder with fewer than 3,000 cases diagnosed in the US every year, characterised by the deposition of amyloid in bodily organs and tissues such as the heart and kidneys.

Ixazomib, the first proteasome inhibitor and first investigational therapy for AL amyloidosis to receive Breakthrough Therapy designation, is currently being assessed alongside dexamethasone in a Phase III trial, TOURMALINE-AL1, in patients with relapsed or refractory forms of the condition. It is also being studied for multiple myeloma.