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MHRA Greenlights First UK Therapy for Congenital Thrombotic Thrombocytopenic Purpura (CTTP)
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved rADAMTS13 (ADZYNMA), the first UK treatment for congenital thrombotic thrombocytopenic purpura (CTTP) in all ages.
What is Congenital TTP (CTTP) Therapy?
CTTP, a rare inherited blood disorder causing blood clots in small blood vessels, affecting blood and oxygen flow to organs. This results in a lower number of platelets, which aid in blood clotting.
Symptoms of Congenital TTP (CTTP)
- Fatigue
- Neurological Symptoms (Headache, Confusion, weakness abnormal sensations and/or seizures
- Abdominal (or) Chest pain
- Breathlessness (due to Anemia)
- Dark Urine
- Bruising and smell bleeds under the skin (small red dots)
On what basis does the medicine had approved?
As per the International Recognition Procedure (IRP), this medicine has approved. It allows MHRA (an executive agency of the Department of Health and Social Care) to take into account the expertise and decision-making of trusted regulatory partners for the benefit of UK patients. The MHRA conducts a thorough evaluation of IRP applications and reserves the right to reject them if the evidence provided is deemed insufficiently robust.
- Takeda UK Ltd, granted on 12 May 2025
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| Area | Address | Phone | Website | |
| Northern and Yorkshire | Yellow Card centre Northern and Yorkshire, Regional Drug and Therapeutics Centre, 16/17 Framlington Place, Newcastle upon Tyne, NE2 4AB | 0191 213 7855 | yccnorthernandyorkshire@nuth.nhs.uk | rdtc.nhs.uk/yellow-card/ |
| Northwest | Yellow Card centre North West, 2nd Floor, 70 Pembroke Place, Liverpool, L69 3GF | 0151 794 8117 | yccnorthwest@rlbuht.nhs.net | www.yccnorthwest.nhs.uk/ |
| West Midlands | Yellow Card centre West Midlands, City Hospital, Dudley Road, Birmingham, B18 7QH | 0121 507 5672 | swb-tr.swbh-team-yccwestmidlands@nhs.net | yccwm.org.uk/ |
| Scotland | Yellow Card centre Scotland, 51 Little France Crescent, Old Dalkeith Road, Edinburgh, EH16 4SA | 0131 242 2919 | YCCScotland@nhslothian.scot.nhs.uk | www.yccscotland.scot.nhs.uk/ |
| Wales | Yellow Card centre Wales, All Wales Therapeutics and Toxicology Centre, Academic Building, University Hospital Llandough, Penlan Road, Penarth, Vale of Glamorgan, CF64 2XX | 029 2184 5831 | CAV_YCCWales@wales.nhs.uk | awttc.nhs.wales/medicines-optimisation-and-safety/yellow-card-centre-wales/ |
| Northern Ireland (NI) | Yellow Card centre Northern Ireland, NI Medicines and Poisons Advice Service (Regional), Pharmacy Department, Knockbracken Clinic (Building 36), Knockbracken Healthcare Park, Belfast Health and Social Care Trust, Saintfield Road, Belfast BT8 8BH | 028 9504 0558 | medicineinfo@belfasttrust.hscni.net | |
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