The Medicines and Healthcare products Regulatory Agency (MHRA) has today (12 May 2025) approved rADAMTS13 (ADZYNMA), the first UK treatment to treat congenital thrombotic thrombocytopenic purpura (CTTP) in patients of all ages.
CTTP is a very rare inherited blood disorder in which blood clots form in small blood vessels throughout the body. These clots can block the flow of blood and oxygen to the body’s organs, which leads to a lower-than-normal number of platelets (components that help the blood to clot) in the blood.
This medicine has been approved through the International Recognition Procedure (IRP). The IRP allows the MHRA to take into account the expertise and decision-making of trusted regulatory partners for the benefit of UK patients…
