Crizanlizumab will become the first new therapy in 20 years to be made available through the U.K.’s National Health Service (NHS) for the treatment of sickle cell disease (SCD). The National Institute for Health and Care Excellence (NICE) recommends crizanlizumab for the prevention of vaso-occlusive crises in patients with SCD aged 16 years or older.
While NICE officials stated there is not enough long-term data to recommend the treatment for routine use at this stage, more than 300 patients per year are expected to receive crizanlizumab via a managed access agreement, which will eventually expand access to more than 450 people.
“We don’t yet know whether the benefits will translate in longer-term outcomes, and we look forward to seeing what the data collected through this managed access agreement will uncover about its benefits for the future,” said Meindert Boysen, PharmD, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.
In 2019, crizanlizumab was approved by the U.S. Food and Drug Administration to reduce the frequency of vaso-occlusive crisis in patients aged 16 and older with SCD.