Biologics

FDA Probes Death Associated With Takeda’s Adzynma

FDA is investigating the death of a pediatric patient with congenital thrombotic thrombocytopenic purpura (cTTP) who developed neutralizing antibodies to the enzyme ADAMTS13 after treatment with Takeda’s Adzynma, a recombinant ADAMTS13 therapy approved in 2023 for the rare, life-threatening blood disorder.

In a new safety communication, the agency says it has received postmarketing reports of neutralizing antibodies in patients treated with Adzynma, including one fatality that “appears to be related” to the medication. No such cases occurred during clinical trials, and the current product labeling does not describe serious or fatal postmarketing outcomes associated with inhibitor development, it says.

According to FDA, the child who died had a medical history of severe allergic reactions to fresh frozen plasma, a standard therapy for cTTP. About 10 months after beginning prophylactic Adzynma treatment, the patient developed neurologic symptoms that worsened, and testing revealed the presence of neutralizing antibodies to ADAMTS13. Current laboratory assays cannot distinguish whether the antibodies targeted the recombinant or naturally occurring enzyme, the agency notes.

FDA says the U.S. prescribing information for Adzynma already includes a warning about the potential development of neutralizing antibodies. The agency says it is actively evaluating the risk and “the need for further regulatory action,” signaling that updates to safety information or stronger warnings may be forthcoming.

cTTP is an ultra-rare genetic disorder that causes blood clots in small vessels, leading to organ damage, low platelet counts, and potentially fatal complications. Adzynma was the first recombinant therapy approved for the condition.

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