Source: United Kingdom – Executive Government & Departments
MHRA approves first UK treatment for congenital thrombotic thrombocytopenic purpura (cTTP)
As with all products, the MHRA will keep its safety under close review.
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.
The MHRA conducts a targeted assessment of IRP applications and retains the authority to reject applications if the evidence provided is not considered sufficiently robust.
As with any medicine, the MHRA will keep the safety and effectiveness of this medicine under close review. Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Notes to editors
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The marketing authorisation was granted on 12 May 2025 to Takeda UK Ltd.
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More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
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The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgments to ensure that the benefits justify any risks.
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The MHRA is an executive agency of the Department of Health and Social Care.
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For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.