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FDA Breakthrough Therapy Designation #58: Takeda Pharmaceutical And Amyloidosis

December 1, 2014

Takeda Pharmaceutical, a global pharmaceutical company located in Osaka, Japan, announces December 1st, that investigational, oral proteasome inhibitor Ixazomib (MLN9708), is granted the FDA Breakthrough Therapy Designation (BTD). The FDA BTD is for relapsed/refractory systemic light-chain (AL) amyloidosis. Ixazomib is the first proteasome inhibitor and the first investigational therapy for AL amyloidosis to receive the FDA BTD.

Ixazomib is being studied in Multiple Myeloma (MM), systemic light-chain (AL) amyloidosis, and other malignancies. Ixazomib receives Orphan Drug Designation (ODD) for MM in both the US (February 2011) and in Europe (2011). Ixazomib receives ODD for systemic light chain AL amyloidosis in both the US (March 2012) and in Europe (2012).

Takeda Pharmaceutical’s Ixazomib Program progressed directly from a Phase I to a Phase III clinical trial ( TOURMALINE-AL), which is currently recruiting and evaluating Ixazomib + Dexamethasone in patients with relapsed/refractory AL amyloidosis. Data used to support the FDA BTD will be presented at the American Society of Hematology (ASH) Annual Meeting, December 6-9, 2014, in San Francisco:

•    Abstract 3450: Long-Term Outcome of a Phase 1 Study of the Investigational Oral Proteasome Inhibitor (PI) Ixazomib at the Recommended Phase 3 Dose (RP3D) in Patients (Pts) with Relapsed or Refractory Systemic Light-Chain (AL) Amyloidosis (RRAL).

“AL amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the U.S. 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. There are no approved treatments in the U.S. or globally for AL amyloidosis, representing a significant unmet medical need.”


Amyloidosis Foundation

Amyloidosis Foundation, “A Guide to Light Chain Amyloidosis”


FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Flickr – Nicholas T – Bows” by Nicholas A. Tonelli from Pennsylvania, USA (Bows) [CC-BY-2.0] | Wikimedia Commons.

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