Takeda touts interim PhIII data of enzyme replacement therapy for rare blood disease
Takeda says in an interim Phase III analysis that its enzyme replacement therapy reduced instances of low blood platelet count for patients with a rare blood clot disorder known as congenital thrombotic thrombocytopenic purpura (cTTP) compared to standard of care plasma-based therapies.
Next, it plans to file for authorization for the drug later this year “broadly — so not just in the US and EMA but also other jurisdictions globally,” Takeda head of rare genetics and hematology Dan Curran told Endpoints News.
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