The approval is based on interim analysis data from a Phase III clinical trial and long-term safety and efficacy results from a continuation study.
The controlled, randomised, open-label, crossover Phase III trial enrolled cTTP patients between 12 and 68 years, including five Japanese patients, to assess the efficacy, pharmacokinetic, safety and tolerability of ADZYNMA.
Takeda research and development head for Japan Yasushi Kajii stated: “The approval of ADZYNMA is an important milestone for people living with cTTP in Japan, who had limited treatment options and now have the first treatment option specifically approved to treat this ultra-rare condition.
“Developing innovative treatments that make a difference in the lives of patients is at the heart of what we do. With this approval, we are proud to support the cTTP community with new possibilities and continue our 70-plus year commitment to the rare disease community.”