Takeda Pharmaceutical Company (TYO: 4502) has secured regulatory approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for its enzyme replacement therapy, Adzynma (apadamtase alfa), to treat congenital thrombotic thrombocytopenic purpura (cTTP) in patients aged 12 and older. cTTP is a rare and potentially life-threatening blood clotting disorder resulting from a deficiency in the ADAMTS13 enzyme.
The approval follows promising interim results from a late-stage clinical trial, which demonstrated that participants receiving Adzynma experienced no acute TTP events by the analysis cutoff. Furthermore, the therapy exhibited a lower rate of treatment-emergent adverse events (TEAEs) compared to a control group treated with plasma-based therapies.
Adzynma is already registered for the same indication in the United States, as well as for TTP in Europe, Japan, and the US, highlighting its growing significance in the treatment landscape for this rare condition.- Flcube.com
