The European Commission has granted a new sickle cell drug authorisation for use in Europe to prevent recurrent sickle cell crises (vaso-occlusive crisis – VOCs). Although this doesn’t currently extend to the UK, it is a great step forward in widening the range of options available for sickle cell patients, which could become available in the UK in the future.  

Global pharmaceutical company Novartis, has announced today that the European Commission has granted Adakveo (crizanlizumab) a conditional marketing authorisation[1] for the prevention of recurrent VOCs in patients with sickle cell aged 16 years and older. 

• Crizanlizumab can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC), or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.
• Crizanlizumab is the first targeted sickle cell disease therapy for prevention of recurrent VOCs available for use in Europe
• Clinical data showed that use of crizanlizumab significantly lowered the median annual rate of VOCs compared to placebo

“Sickle cell is a chronic, life-long condition characterised by episodes of pain that can last from a few minutes to several weeks, though most last five to seven days on average [2] . Pain crises are unpredictable and have a significant impact on an individual’s long term health and quality of life; often resulting in hospital admissions,” says John James OBE, Chief Executive, Sickle Cell Society. “There is an urgent need for increased awareness and understanding of the condition, as well as the development of tools and therapies that enable patients to better manage their disease and have more pain-free days.”

“Nine out of ten people living with sickle cell disease in the UK experienced at least one VOC in the past year, with a third of these crises leading to hospitalisation, highlighting the significant unmet need amongst these patients [3],” said Mari Scheiffele, General Manager, Novartis Oncology UK & Ireland. “Adakveo is the first targeted therapy for prevention of recurrent VOCs and the first new treatment for sickle cell disease in over twenty years. We are working closely with UK regulatory bodies to bring UK patients access to this treatment.”

For a long time, many sickle cell patients have had to rely on just one drug, hydroxycarbamide/hydroxyurea. A new drug being licensed in Europe, which focuses on reducing painful crises is exciting news and shows that more research is being invested in sickle cell. Hopefully, we will continue to see more options becoming available for sickle cell patients in the UK and across the world. 

You can learn more here: https://www.novartis.com/news/media-releases/novartis-sickle-cell-medicine-adakveo-approved-europe-prevent-recurrent-vaso-occlusive-crises

1. Conditional Marketing Authorisation – the approval of a medicine that address unmet medical needs of patients on the basis of less comprehensive data than normally required. The available data must indicate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data in the future. [https://www.ema.europa.eu/en/glossary/conditional-marketing-authorisation Acessed: 30/10/2020]
2. Sickle Cell Society. About Sickle Cell Disease. Available from: https://www.sicklecellsociety.org/about-sickle-cell/ [Accessed August 2020]
3. Inusa B, et al. UK Patient perceptions on the symptomatic and emotional burden of vaso-occlusive crises resulting from Sickle cell disease. British Journal of Haematology. Abstract BSH2020-PO-079A