Takeda Pharmaceuticals’ Genetic Disease Therapy Under FDA Probe After Pediatric Patient Dies

The death of a pediatric congenital thrombotic thrombocytopenic purpura patient appears to be related to Adzynma, the FDA said.
Takeda Pharmaceutical Company, building exterior with company sign, 500 Kendall Street, Cambridge, Massachusetts, USA. (Photo by: Plexi Images/GHI/UCG/Universal Images Group via Getty Images)
Takeda Pharmaceutical Company, building exterior with company sign, 500 Kendall Street, Cambridge, Massachusetts, USA. (Photo by: Plexi Images/GHI/UCG/Universal Images Group via Getty Images)
Profile Image
Anan Ashraf·Stocktwits
Published Nov 21, 2025   |   9:41 AM EST
Share
·
Add us onAdd us on Google

The U.S. Food and Drug Administration (FDA) said on Friday that it is investigating the death of a patient treated with Adzynma.

Adzynma, manufactured by Takeda Pharmaceuticals (TAK), is approved for patients with congenital thrombotic thrombocytopenic purpura (cTTP), also known as Upshaw-Schulman syndrome. It is a rare inherited disorder characterized by a severe deficiency of the ADAMTS13 enzyme.

The death of a pediatric cTTP patient appears to be related to Adzynma, the FDA said. The FDA is now investigating the risk of development of neutralizing antibodies with serious, including life-threatening or fatal, outcomes following treatment with Adzynma. 

Get updates to this developing story directly on Stocktwits.

Share
·
Add us onAdd us on Google
Read about our editorial guidelines and ethics policy