Novartis' Adakveo (crizanlizumab) has hit a setback having failed to gain acceptance by the National Institute for Health and Care Excellence (NICE).
The cost watchdog has not recommended the drug's NHS use, within its marketing authorisation, for preventing recurrent sickle cell crises (vaso-occlusive crises) in people aged 16 or over with sickle cell disease.
Current treatment to prevent sickle cell crises includes hydroxycarbamide (or hydroxyurea), which is taken as a tablet, or regular blood transfusions.
Adakveo is injected into the vein in people aged 16 or over either as monotherapy or alongside hydroxycarbamide.
According to NICE, clinical evidence suggests that people taking the drug have fewer sickle cell crises in a year than if they do not have any treatment. “However, these results are not certain because the trial was short and included only a small number of people on the licensed dose of the drug,” it noted.
Furthermore, the Institute also said there is also uncertainty about the cost-effectiveness estimates, which it believes are likely substantially higher than what is normally considered a cost-effective use of NHS resources.