FDA Approves Takeda’s Treatment for Rare Blood Clotting Disorder

Pharmaceutical Executive November 9, 2023
Pharmaceutical Executive Editorial Staff

Adzynma is the first approved genetically engineered protein product for the treatment of patients with congenital thrombotic thrombocytopenic purpura.

The FDA has approved Takeda’s Adzynma as the first genetically engineered protein medication for the preventative treatment or for on-demand enzyme replacement therapy in patients with congenital thrombotic thrombocytopenic purpura (cTTP).¹

The FDA granted the application for Adzynma with a Rare Pediatric Disease Priority Review Voucher, as well as Priority Review, Fast Track, and Orphan designations. cTTP affects fewer than 1,000 individuals in the United States.¹

“The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases,” said FDA Director of the Center for Biologics Evaluation and...

Today's Sponsors

Today's Sponsor