NICE recommends approval of Cablivi for thrombotic thrombocytopenic purpura.- Sanofi

Sanofi announced 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. The NICE recommendation of caplacizumab is based on the data from the HERCULES Phase III study. Caplacizumab is the first treatment specifically approved for aTTP in 30 years and the only therapy approved for people experiencing an aTTP episode.The study compared the efficacy and safety of caplacizumab vs placebo, in addition to the standard of care, in 145 patients with aTTP. 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).>