SMC recommends Cablivi®▼ (caplacizumab) for adults with aTTP, a rare life-threatening blood-clotting disorder 
- Acquired thrombotic thrombocytopenic purpura (aTTP) is a medical emergency,2 where sudden complications like heart attack3 and kidney failure4 can occur without warning5
- Whilst TTP only affects 15 people per year in Scotland,1 one in two will die unless they can get to a specialist centre;6 this rises to 90% for those who are unable to access treatment7
- Caplacizumab is the first new therapy developed to treat aTTP in 30 years and the only therapy approved for adults experiencing an episode8,9
READING, ENGLAND – 7th September 2020 - Sanofi announced today that the Scottish Medicines Consortium (SMC) has recommended Cablivi® (caplacizumab) for adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.1
Appraised by the SMC as an orphan indication, aTTP is an ultra-rare, complex and potentially life-threatening blood clotting disorder, that can cause neurologic symptoms and result in severe organ damage and death.2,10,11 Following the first episode of aTTP, 30-50% of people relapse12 and without treatment, up to 90% of people will experience associated long-term health complications or death.13
Caplacizumab is the first and only specific therapy approved in conjunction with plasma exchange and immunosuppression for the treatment of adults experiencing an episode of aTTP.8,9
“Caplacizumab is a promising new treatment that now fills an unmet need,” says Marie Scully, Professor of Haematology and Consultant at University College London Hospitals. “This is a very positive development and I am pleased that the SMC has recognised both the level of unmet need for people with aTTP and the evidence which supports caplacizumab as a viable treatment option.”
Prior to caplacizumab, there has not been a new option in the treatment of acute aTTP since 1991 when plasma exchange (the current standard of treatment) was introduced.14 Patients undergoing plasma exchange treatment are required to spend up to three sessions in the hospital each day for about two weeks.6
The SMC carried out an evaluation of the clinical and cost-effectiveness of caplacizumab and based its advice on data from the HERCULES Phase III clinical study.1 The study compared the efficacy and safety of caplacizumab vs placebo, both during plasma exchange, in 145 patients with aTTP.15
Results showed that caplacizumab, compared with placebo, in addition to plasma exchange, decreased the time for platelets, damaged by the disorder, to recover (placebo = 4.50 days and caplacizumab = 2.95 days) and reduced the risk of death, recurrence of TTP or formation of major clots in blood vessels (placebo = 36% and caplacizumab = 9%).15
Reduction was also seen in the use of plasma exchange in people treated with caplacizumab (placebo = 9.4 days and caplacizumab = 5.8 days), as well as a shorter stay in the intensive care unit (placebo = 9.7 days and caplacizumab = 3.4 days) and hospital (placebo = 14.4 days and caplacizumab = 9.9 days).15
“Sanofi is committed to improving the lives of people who are unpredictably afflicted with aTTP which presents as a life-threatening medical emergency,” says Fleur Chandler, Head of Market Access at Sanofi UK & Ireland. “Today’s acceptance represents an important milestone in the history of aTTP and one which we hope will improve the future for many people living with the condition. We are pleased that we have been able to work collaboratively with the SMC to make caplacizumab available for patients in need.”
About Cablivi (caplacizumab)
Cablivi (caplacizumab) uses innovative nanobody technology to provide patients with aTTP rapid protection from blood clots in small blood vessels.8,16 Blood clots lead to a very low platelet count, destruction of red blood cells and restricted blood supply to parts of the body.2 When used in conjunction with plasma exchange and immunosuppression, caplacizumab, compared to placebo, helps to normalise the platelet count faster, reduce the risk of acute thrombotic complications, disease recurrence, lack of treatment response and death.15
Caplacizumab was licenced for use in adults in Europe on 31st August 2018.17
About the HERCULES trial
HERCULES was a Phase III, double-blind, placebo-controlled, randomised trial to evaluate the efficacy of caplacizumab, compared to placebo, in addition to plasma exchange, in more rapidly restoring normal platelet counts as a measure of prevention of further microvascular thrombosis.18
145 patients with aTTP were enrolled and randomly assigned to receive caplacizumab (10mg intravenously followed by 10mg daily subcutaneously) or placebo during plasma exchange (PEX) and for 30 days thereafter.15 The primary outcome was the time to normalisation of the platelet count, with discontinuation of daily plasma exchange within five days thereafter.15 Key secondary outcomes included a composite of aTTP-related death, recurrence of aTTP, or a thromboembolic event during the trial treatment period; recurrence of aTTP at any time during the trial; refractory aTTP; and normalisation of organ-damage markers.15
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
Media Relations Contacts
Tel. +44 (0) 1183 543 485
Mob. +44 (0) 7935 503 416
Tel.: +44 (0) 7971 743 565
1. Caplacizumab 10mg powder and solvent for solution for injection (Cablivi®). SMC2266. Scottish Medicines Consortium (SMC). Available at: https://www.scottishmedicines.org.uk. Last accessed. September 2020.
2. Joly BS, Coppo P, Veyradier. Thrombotic thrombocytopenic purpura. Blood. 2017; 129:2836-2846.
3. Yves Benhamou, et al. Cardiac troponin-I on diagnosis predicts early death and refractoriness in acquired thrombotic thrombocytopenic purpura. Journal of Thrombosis and Haemostasis. 2015;13(2). Available at: doi.org/10.1111/jth.1279 . Last accessed. September 2020.
4. Han-Mou Tsai. The kidney in thrombotic thrombocytopenic purpura. Minerva Medical. 2007; 98(6):731–747. Available at: www.ncbi.nlm.nih.gov/pmc/articles/PMC2430013/. Last accessed. September 2020.
5. Marie Scully et al. Management of thrombotic thrombocytopenic purpura: current perspectives. 2014; 2014(5). Available at: doi.org/10.2147/JBM.S46458. Last accessed. September 2020.
6. NHS England. Integrated Impact Assessment Report for Service Specifications. 2018. Available at: https://www.engage.england.nhs.uk/consultation/thrombocytopenic-purpura/user_uploads/thrombotic-thrombocytopenic-purpura-impact-assessment.pdf. Last accessed: September 2020.
7. Marie Scully, et al. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br Journal of Haematology. 2012;158(3):323–35. Available: doi.org/10.1111/j.1365-2141.2012.09167.x. Last accessed: September 2020.
8. EMA. Cablivi Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/cablivi-epar-product-information_en.pdf. Last accessed: September 2020.
9. Morrison, C. Nanobody approval gives domain antibodies a boost. Nature Reviews Drug Discovery. 2019. 18:485-487.
10. Thejeel B, Garg AX, Clark WF, et al. Long‐term outcomes of thrombotic microangiopathy treated with plasma exchange: A systematic review. American Journal of Hematology. 2016; 91:623-360.
11. Peyvandi F, Scully M, Hovinga JA, et al. Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. New England Journal of Medicine. 2016; 374:511-522.
12. Scully, M and Goodship, T. How I treat thrombotic thrombocytopenic purpura and atypical haemolytic uraemic syndrome. British Journal of Haematology. 2014; 164:759–766
13. Grall M, Azoulay E, et al. Thrombotic Thrombocytopenic Purpura Misdiagnosed As Autoimmune Cytopenia: Causes of Diagnostic Errors and Consequence on Outcome. Experience of the French Thrombotic Microangiopathies Reference Centre. American Journal of Hematology. 2017; 92(4):381-387.
14. Johanna A, Hovinga K, Vesely SK, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010; 115(8):1500-1511.
15. Scully M et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019. 380:335-46.
16. Ablynx. Understanding Nanobodies. Available at: https://www.ablynx.com/technology-innovation/understanding-nanobodies. Last accessed: September 2020
17. EMA. Cablivi. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/cablivi. Last accessed: September 2020.
18. ClinicalTrials.gov. Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES). Available at: https://clinicaltrials.gov/ct2/show/NCT02553317. Last accessed: September 2020.