Wednesday, March 27, 2024
Takeda (TSE: 4502/NYSE:TAK) has announced the approval of ADZYNMA (apadamtase alfa/cinaxadamtase alfa) by the Japanese Ministry of Health, Labour and Welfare for treating congenital thrombotic thrombocytopenic purpura (cTTP) in individuals aged 12 and above. cTTP, an extremely rare clotting disorder, results from a deficiency in the ADAMTS13 enzyme and can lead to severe complications if left untreated, with mortality rates exceeding 90% for acute events.
Yasushi Kajii, Head of R&D Japan Region at Takeda, emphasized the significance of this approval, marking a crucial advancement for cTTP patients in Japan who previously had limited treatment options. ADZYNMA, the first and only recombinant ADAMTS13 protein approved for cTTP, aims to address this unmet medical need by replacing the deficient enzyme.
The approval is based on comprehensive evidence from a Phase 3 trial (281102 NCT03393975), including data from Japanese patients, and is further supported by long-term safety and efficacy data from a continuation study (TAK-755-3002 NCT04683003). Notably, during the study, no acute TTP events occurred in patients receiving ADZYNMA prophylactic treatment, contrasting with one event in patients receiving plasma-based therapies.
Regarding safety, the incidence of treatment-related adverse events was significantly lower in the ADZYNMA group compared to those receiving plasma-based therapy, indicating a favorable safety profile for ADZYNMA. Moreover, Takeda stated that this approval doesn't alter its fiscal forecast for the year ending March 31, 2024 (FY2023).
ADZYNMA, also approved by the U.S. FDA, offers both prophylactic and on-demand treatment options for cTTP patients, providing flexibility in managing this debilitating condition. Its recommended dosage and administration have been outlined, catering to both prophylactic and acute treatment needs.
CTTP is a challenging condition associated with life-threatening complications and chronic symptoms. ADZYNMA's approval marks a significant step forward in addressing the unmet needs of cTTP patients, reflecting Takeda's commitment to advancing treatments for rare diseases.
Source: takeda.com
