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.

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.<