NICE recommends Cablivi®▼ (caplacizumab) for people with acquired thrombotic thrombocytopenic purpura, a rare blood-clotting disorder[i]

NICE recommends Cablivi^®▼ (caplacizumab) for people with acquired thrombotic thrombocytopenic purpura, a rare blood-clotting disorder[i]

• Every year, acquired thrombotic thrombocytopenic purpura (aTTP) affects approximately six people per million in the UK[ii]
• aTTP presents as a medical emergency;[iii] Up to 50% of people will die unless they can get specialist treatment,[iv] rising to up to 90% for those who are unable to access treatment at all2
• Caplacizumab is the first treatment specifically approved for aTTP in 30 years and the only therapy approved for people experiencing an aTTP episode[v]^,[vi] 

READING, ENGLAND – 12^th November 2020 - Sanofi announced today that the National Institute for Health and Care Excellence (NICE) has recommended Cablivi^®  (caplacizumab) with plasma exchange and immunosuppression, as an option for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP) in adults and in young people aged 12 years and over who weigh at least 40 kg.¹

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.^{3,[vii],[viii]} Following the first episode of aTTP, 30-50% of people relapse[ix] and without treatment, up to 90% of people will experience associated long-term health complications or death.^3,7

“aTTP is a harrowing condition that requires rapid diagnosis and treatment – often in intensive care,” says Marie Scully, Professor of haematology and consultant at University College London Hospitals. “Today’s decision marks the availability of a critical treatment option for people who experience this rare but devastating disease”

This decision acknowledges the importance of caplacizumab as a treatment option in addressing the unmet needs of people with aTTP. Until now, those suffering an episode of aTTP could only rely on plasma exchange and immunosuppression.

“Until now, treatment for those with TTP has been limited, so we welcome NICE’s recommendation of caplacizumab as a much-awaited addition to treatment options for patients with TTP,” says Jo McIntyre, Founder, TTPNetwork, the UK patient led support group. “TTP has lasting physical and psychological impact on individuals and their families and this recommendation has provided another option.”

The NICE recommendation of caplacizumab is based on the data from the HERCULES Phase III study. The study compared the efficacy and safety of caplacizumab vs placebo, in addition to the standard of care, in 145 patients with aTTP.⁸

Results showed that caplacizumab, compared with placebo, in addition to standard of care, decreased the median time for platelets to recover to normal levels (median; placebo = 2.88 days and caplacizumab = 2.69 days; p-value = 0.01) and reduced the risk of death, recurrence of TTP or formation of major clots in blood vessels (placebo = 49% and caplacizumab = 12%; p-value <0.001).⁸

As a treatment for an ultra-rare disease, caplacizumab was initially to be considered within the Highly Specialised Technology (HST) process but was assessed under the Single Technology Appraisal (STA) process. The STA does not consider the concerns around long-term follow-up and management, which are a well-known issue in evaluating medicines for ultra-rare diseases.

“We are pleased that NHS patients will be granted access to this treatment in the future. It has, however, been a long-awaited result, delaying access for those most in need, and one which could have been simplified if caplacizumab had been assessed under the Highly Specialised Technology process. Throughout this time, we have remained committed to aTTP patients through the free supply scheme we have had in place for over two years. We now hope that the forthcoming NICE Methods Review and Innovative Drugs Fund consultation will fix the ongoing barriers for patients,” says Deborah Lough, Head of Rare Blood Disorders UK & Ireland Sanofi Genzyme.

[i] NICE Caplacizumab for treating an episode of acquired thrombotic thrombocytopenic purpura [ID1185].

 Last accessed November 2020.

[ii] Scully M 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.

[iii] Joly BS, Coppo P, Veyradier. Thrombotic thrombocytopenic purpura. Blood. 2017; 129:2836-2846

[iv] 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: November 2020.

[v] Morrison, C. Nanobody approval gives domain antibodies a boost. Nature Reviews Drug Discovery. 2019. 18:485-487.

[vi] 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: November 2020.

[vii] 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.

[viii] Peyvandi F, Scully M, Hovinga JA, et al. Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. New England Journal of Medicine. 2016; 374:511-522.

[ix] Scully, M and Goodship, T. How I treat thrombotic thrombocytopenic purpura and atypical haemolytic uraemic syndrome. British Journal of Haematology. 2014; 164:759–766

[x] Ablynx. Understanding Nanobodies. Available at: https://www.ablynx.com/technology-innovation/understanding-nanobodies/. Last accessed: November 2020.

[xi] EMA. Cablivi. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/cablivi. Last accessed: November 2020.

[xii] 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: November 2020.