cTTP, an ultra-rare blood clotting disorder, presents life-threatening acute events and debilitating chronic symptoms. Untreated acute TTP events carry a mortality rate exceeding 90%. ADZYNMA aims to alleviate symptoms associated with cTTP, including thrombocytopenia, microangiopathic hemolytic anemia, headache, and abdominal pain.

Yasushi Kajii, Head of R&D Japan Region at Takeda, emphasized the significance of ADZYNMA's approval in Japan, stating it provides a crucial treatment option for those with cTTP, who previously had limited choices. Kajii underscored Takeda's commitment to innovation in rare disease treatments and its dedication to improving patients' lives.

The approval is grounded on comprehensive evidence, including interim analysis of efficacy, pharmacokinetic, safety, and tolerability data from a Phase 3 trial involving cTTP patients aged 12-68. Notably, during the trial's controlled comparison periods, no acute TTP events were reported in patients receiving ADZYNMA prophylactic treatment, whereas one event occurred in a patient receiving plasma-based therapies.

Treatment-emergent adverse events (TEAEs) assessed as treatment-related were significantly lower in patients receiving ADZYNMA compared to plasma-based therapy recipients. Common TEAEs included constipation, abnormal ADAMTS13 activity, headache, pruritus, and hypertension.

Takeda's approval of ADZYNMA in Japan does not impact its consolidated forecast for the fiscal year ending March 31, 2024 (FY2023).