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NICE recommends Sanofi’s Cablivi for ultra-rare acute thrombotic thrombocytopenic purpura

Published on 12/11/20 at 12:43pm

Sanofi’s Cablivi (caplacizumab) has received recommendation from NICE for routine NHS use in England and Wales for the treatment of acute episodes of the acquired ultra-rare blood-clotting condition thrombotic thrombocytopenic purpura (TTP), when used in combination with plasma exchange and immunosuppression.

The decision was reached in part because of the severe unmet need for treatment options among the aTTP population, with the only option being plasma exchange and immunosuppression alone. The recommendation relates to patients over the age of 12 who weigh at least 40 kilograms.

Phase 3 data drawn from 145 aTTP patients showed that caplacizumab, in combination with standard of care, reduced median platelet recovery time to 2.69 days compared to 2.88 days with placebo plus standard of care. The combo also reduced risk of death, disease recurrence or major clot formation by 37% to 12%, compared to 49% with the placebo combo.

“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,” commented Jo McIntyre, Founder at patient support group TTPNetwork. “TTP has lasting physical and psychological impact on individuals and their families and this recommendation has provided another option.”

The condition is a potentially fatal one that is characterised by neurologic symptoms and often leading to severe organ damage. Between 30% and 50% of patients relapse following their first episode, and as much as 90% will experience long-term health complications or death without adequate treatment.

“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,” remarked Deborah Lough, Head of Rare Blood Disorders UK & Ireland Sanofi Genzyme.

“Throughout this time, we have remained committed to aTTP patients through the free supply scheme we have had in place for over two years,” she added. “We now hope that the forthcoming NICE Methods Review and Innovative Drugs Fund consultation will fix the ongoing barriers for patients.” 

Matt Fellows

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