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

• After about 10 months of starting treatment with Adzynma, the presence of neutralizing antibodies was identified in the patient who died, 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.
• The agency noted that neutralizing antibodies were not reported in clinical trials.

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

Adzynma, a man-made enzyme therapy manufactured by Takeda Pharmaceuticals (TAK), is approved for adult and pediatric 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.

Findings And Further Probe

Before starting Adzynma treatment, the patient had severe allergic reactions to fresh frozen plasma, the FDA noted. After about 10 months of starting treatment with Adzynma, the presence of neutralizing antibodies was identified.

However, the agency also noted that current assays cannot differentiate the neutralizing antibodies to man-made enzyme therapy from the neutralizing antibodies to the body’s own enzymes. The agency also added that it has received other reports of neutralizing antibodies in patients treated with the drug since approval.

The FDA is now investigating the risk of development of neutralizing antibodies with serious, including life-threatening or fatal, outcomes following treatment with Adzynma. The agency noted that neutralizing antibodies were not reported in clinical trials and that the current labeling does not include the risk of serious and fatal outcomes.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around TAK stayed within the ‘neutral’ territory over the past 24 hours, while message volume stayed at ‘low’ levels.

TAK stock has gained 8% this year and about 7% over the past 12 months.

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