Takeda’s Adzynma approved for prophylactic and on-demand treatment of cTTP

Takeda announced at the end of last month (November 2023) that it had been approved by the US Food and Drug Administration (FDA) on November 9, 2023.

cTTP is a very rare, complex condition that can present as an acute life-threatening disorder requiring prompt diagnosis, early referral, and effective immediate and ongoing management in a center with comprehensive provision and a multi-discipline approach.

Adzynma is the first and only recombinant ADAMTS13 enzyme replacement therapy for people with cTTP designed to address the root cause of the condition, an ADAMTS13 enzyme deficiency.

On-demand treatment for cTTP

Adults and children in the US with cTTP can now access Adzynma for prophylactic and on-demand treatment.

“For nearly a century, people impacted by this debilitating, ultra-rare condition have been without a treatment option specifically indicated for their disease,” said Cheryl Schwartz, senior vice president of, Rare Disease Business Unit at Takeda.

“Patients have waited too long, and so it is with great pride that we announce the availability of the first and only FDA-approved treatment for patients with cTTP to directly address their ADAMTS13 enzyme deficiency.”

Significant milestone for cTTP community

The ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations. These can include thrombocytopenia, microangiopathic hemolytic anemia, headache, and abdominal pain. When left untreated, acute TTP events have a mortality rate of more than 90%.

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