Takeda's enzyme therapy approved in Europe for rare blood disease

The European Commission has granted approval to Takeda's enzyme replacement therapy (ERT), Adzynma, for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) in both children and adults. This rare disease leads to the formation of blood clots and this authorization marks the first ERT approval for cTTP in the region. This decision follows closely on the heels of the drug’s approval by the FDA just under a year ago and a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in May.

Ricardo Marek, who presides over Takeda’s Europe and Canada business unit, stated that Adzynma is specifically designed to tackle the fundamental cause of cTTP. He emphasized that patients suffering from this condition are still at risk of life-threatening acute episodes and enduring chronic symptoms. Adzynma is a purified, recombinant form of the ADAMTS13 enzyme, intended to replace the deficient enzyme levels in cTTP patients.

For prophylactic ERT, Adzynma is administered intravenously once every two weeks, aiming to decrease the likelihood of disease symptoms. It can also be given once daily for on-demand ERT when patients experience acute events. The approval of Adzynma was substantiated by data from a global Phase III clinical trial. In this trial, none of the patients who received Adzynma experienced an acute TTP event, while one patient in the control group, which received plasma-based therapy, did encounter such an event. Additionally, within the experimental group, only one patient had a subacute TTP event, compared to seven events in six patients on plasma-based therapy.

Adzynma also showed a superior safety profile compared to the control therapy. The most commonly observed adverse reactions were generally mild, including headache, diarrhea, dizziness, nausea, and migraine.

This approval by the European Commission represents a significant milestone in the treatment of cTTP, offering a new therapeutic option for patients suffering from this debilitating disease.