Takeda scores a 'breakthrough' tag for PhIII rare disease drug

With its bottom line getting squeezed and big budget cuts looming, Takeda and its new president, Christophe Weber, issued some upbeat news today for a change. The FDA has branded its top late-stage drug prospect ixazomib (MLN9708)--a multiple myeloma treatment designed to succeed the top-selling blockbuster Velcade--as a "breakthrough" therapy deserving VIP status with regulators.

The FDA handed out the breakthrough designation for the company's work on systemic light-chain (AL) amyloidosis, a condition linked to multiple myeloma in which amyloid builds up and damages organs in the body. Takeda has the most advanced program in the clinic for amyloidosis, jumping from Phase I straight into Phase III, but the Irish biotech Prothena ($PRTA) has a drug in earlier-stage development for the same disease.

According to Takeda, AL amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the U.S. every year.

Takeda has had some success in the recent past, most notably the approval of vedolizumab for inflammatory bowel disease. But it's also had late-stage failures for TAK-875, or fasiglifam, as well as their top cancer contender at their Cambridge, MA-based cancer subsidiary Millennium. Those setbacks helped trigger a plan to carve $1 billion out of its budget and lay off thousands.

"This Breakthrough Therapy designation is also an important recognition of the strength of our oncology development program and our commitment to extend proteasome inhibition to conditions that have not traditionally been the focus of research and development," said Dr. Michael Vasconcelles, who heads up the oncology therapeutic area unit for Takeda, in a statement. "We would like to acknowledge and thank the patients, study investigators, and institutions whose participation in this program have made this milestone possible."

- here's the release

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