Sickle Cell Disease



AOFAC Foundation’s response to European Medicines Agency (EMA) and Novartis updates on Crizlanlizumab – 30/01/2023

As you may be aware, Crizanlizumab (Adakveo), a new disease-modifying treatment for sickle cell anaemia was recommended the by National Institute of Clinical Excellence ( NICE) and NHS England on a Managed Access Agreement. 

 Under the Managed Access Agreement, Crizanlizumab was recommended to eligible patients to collect more data about the treatment.

Following the release of the preliminary findings from the ongoing worldwide clinical study called STAND (NCT038147460), the results indicate that there is no statistically significant difference between Crizanlizumab 5mg/kg and Crizanlizumab 7.5mg/kg and placebo in impacting the rates of sickle cell crises leading to a healthcare visit. However, these findings are inconsistent with previous trial results from the SUSTAIN (NCT01895361) trial. As a result of this regulatory bodies such as the European Medicines Agency (EMA) will undertake a review of Crizanlizumab to evaluate the impact of these contrasting results on its currently authorised use.

It is important to note that the preliminary results do not suggest new safety concerns with crizanlizumab.

If you have any concerns, we recommend that you speak to your Consultant Haematologist who will determine the best option for each patient depending on their individual situation.

We will continue to provide more information when available.