The UK’s National Institute for Health and Care Excellence (NICE) has given a green light to Sanofi’s Cablivi for acquired thrombotic thrombocytopenic purpura (aTTP) – a rare and potentially life-threatening blood clotting disorder.

The new NICE guidance applies to the treatment of an acute episode of aTTP in adults and in young people aged 12 years and over who weigh at least 40kg.

The decision to recommend Cablivi (caplacizumab) is based on data from the phase III HERCULES study, which compared the efficacy and safety of Cablivi compared to placebo, plus standard of care, in 145 patients with aTTP.

The results from that study showed that Cablivi in addition to standard of care, when compared to placebo, decreased the median time for platelets to recover to normal levels and reduced the risk of death, as well as recurrence of TTP or formation of major clots in blood vessels.

Although Cablivi is an ultra-rare disease treatment and was initially set to be considered with NICE’s Highly Specialised Technology (HST) process, it was finally assessed under the Single Technology Appraisal (STA) process.

“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 Cablivi had been assessed under the Highly Specialised Technology process,” said 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. We now hope that the forthcoming NICE Methods Review and Innovative Drugs Fund consultation will fix the ongoing barriers for patients,” she added.