The first treatment for sickle cell disease in over 20 years, Novartis' Adakveo®▼ (crizanlizumab) receives NICE recommendation for preventing recurrent vaso-occlusive crises

- Significant step forward in the commitment to advancing health equity for thousands of people living with sickle cell disease (SCD) in the UK

- Treatment recommended by the National Institute for Health and Care Excellence (NICE) for people aged 16 or older under a Managed Access Agreement (MAA) in partnership with NHS England

- Partnership will see real-world data collected via the National Haemoglobinopathy Register, helping make the UK a centre of excellence for SCD care 

- Vaso-occlusive crises (VOCs) disrupt patients' lives physically, socially, and emotionally, and can increase risk of organ damage, costly hospitalisations and early death^[1]

LONDON, Oct. 5, 2021 /PRNewswire/ -- Novartis is pleased to announce that eligible patients in England and Wales will soon have routine access to Adakveo^®▼ (crizanlizumab) under a Managed Access Agreement (MAA). The news comes as the National Institute for Health and Care Excellence (NICE), published the Final Appraisal Determination (FAD) recommending crizanlizumab as an option for preventing recurrent sickle cell crises (two or more vaso-occlusive crises, VOCs, in a year, managed at home or in hospital) in people aged 16 or older with sickle cell disease (SCD).^[2] Access to this innovative treatment not only helps with SCD but also demonstrates a commitment to supporting health equity for a community that has been historically underserved, without any new treatment options for more than 20 years, and disproportionately impacted by COVID-19.

"Sickle cell crises are much more than unpredictable, painful events: recurrence is a constant worry that pervades every aspect of daily life," commented John James OBE, Chief Executive, Sickle Cell Society. "For the thousands of people living with this complex and life-long disease in this country, this decision offers hope of a life with less disruption and stress from their condition, as well as reducing the associated costs to the NHS."

VOCs are one of the main symptoms of SCD.^[3],[4] The sickling of red blood cells can result in the cells sticking together, along with increased adhesion to other blood cells and blood vessel walls. This can cause blockages in smaller blood vessels and trigger episodes of acute pain (crises).^[4],[5] Sickle cell pain crises disrupt patients' lives physically, socially, and emotionally – and can worsen into acute and long-term complications.^[6] People living with SCD suffer an increased risk of high blood pressure, eyesight loss, and kidney and urinary problems, along with long-term damage to internal organs, such as kidneys, liver, spleen, heart and lungs.^[3] In addition, SCD can have a significant impact on an individual's ability to engage in normal life, limiting career and educational opportunities, along with associated mental health challenges.^[7]

The decision follows close consultation between NICE, NHS England and Novartis, as well as professional and patient/carer groups across the United Kingdom. By adopting a collaborative approach – including collecting prospective real-world data via the National Haemoglobinopathy Register (NHR) – this decision clears the way for patients with SCD in England to be able to access crizanlizumab. In addition to collecting valuable real world evidence which has historically been lacking in this disease area, the partnership agreement between Novartis, NHR, NICE and NHS England aims to identify and address any inequality of uptake, supporting long-term access to treatment and helping to make the UK a centre of excellence for SCD care. Securing a positive decision and MAA for crizanlizumab demonstrates how this kind of partnership can enable patients with a huge unmet need to gain early access to innovative treatments, and can help inform the implementation of the recently-announced Innovative Medicines Fund.^[8]

"Vaso-occlusive crises are acute episodes of pain that often last for days, and have a substantial impact on the quality of life of people living with sickle cell disease. These episodes can also lead to organ damage and significant long-term complications," added Professor Baba Inusa, Chair of the National Haemoglobinopathy Panel, Professor of paediatric haematology at King's College London and lead consultant for paediatric haemoglobinopathies, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust. "Today's decision is an important milestone for the medical community: for the first time in 20 years, there will now be a new treatment regime that we can offer this community, which has gone for so long without additional support in managing their condition."

"Today's announcement reinforces Novartis' commitment to exploring new approaches and partnerships that deliver innovative treatments to patients living with rare diseases," said Kees Roks, Head of Region Europe, Novartis Oncology. "At Novartis, we are proud to work with the NHS and the extended healthcare network  in the UK to identify and deliver solutions for those patients most in need, no matter their background or location. This important partnership agreement enabling patient access to crizanlizumab is the result of positive collaboration between Novartis, NICE, NHS England and the sickle cell community."

Notes to Editors

About sickle cell disease

Sickle cell disease is a complex, genetic blood disorder that is inherited from both parents.^[3] It is estimated that there are currently 15,000 people with the condition living in the UK.^[3] While sickle cell disease mainly affects people of African or African-Caribbean origin, the sickle gene is found in all ethnic groups.^[9]

About Adakveo^® (crizanlizumab)

Adakveo^® (crizanlizumab) – previously known as SEG101 – is licensed for the prevention of recurrent VOCs in sickle cell patients aged 16 years and older. It 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. It is the first and only targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion in sickle cell disease. By binding to P-selectin on the surface of the activated endothelium and platelets, crizanlizumab blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes.^[5],[10]

Crizanlizumab was granted conditional Marketing Authorisation by the European Medicines Agency in October 2020.^[2]

About Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we strive to use innovative science and digital technologies to create treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world.

In the UK, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis. Since 2014, Novartis has invested over £200 million in R&D and is a leading sponsor of clinical trials in the UK. For more information, please visit www.novartis.co.uk.

Novartis UK is on Twitter. Sign up to follow @NovartisUK at www.twitter.com/novartisuk.

References

1. Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.
2. Crizanlizumab SmPC, 2021.
3. Sickle Cell Society. About Sickle Cell Disease. Available from: https://www.sicklecellsociety.org/about-sickle-cell/ [Accessed October 2021].
4. Kenny T and Tidy C. Sickle Cell Disease and Sickle Cell Anaemia patient information leaflet. 2015. Available at: https://www.stgeorges.nhs.uk/wp-content/uploads/2015/03/SCD-information-from-patient-co-uk.pdf [Accessed October 2021].
5. Kaul DK, Finnegan E, Barabino GA. Sickle red cell-endothelium interactions. Microcirculation. 2009 Jan;16(1):97-111.
6. Adegbola M, et al. Voices of adults living with sickle cell disease pain. J Natl Black Nurses Assoc. 2012;23(2):16-23.
7. Osunkwo I, Andemariam B, Inusa B, et al. Management Strategies and Satisfaction Levels in Patients With Sickle Cell Disease: Interim Results From the International Sickle Cell World Assessment Survey (SWAY). presented at: The American Society of Hematology Annual Meeting; December 7-10, 2019; Orlando, FL.
8. NHS England, 2021. NHS England announces new Innovative Medicines Fund to fast-track promising new drugs. [online] Available at: https://www.england.nhs.uk/2021/07/nhs-england-announces-new-innovative-medicines-fund-to-fast-track-promising-new-drugs/ [Accessed October 2021].
9. Gillis, VL et al. Management of an acute painful sickle cell episode in hospital: summary of NICE guidance. BMJ. 2012 Jun 27;344:e4063. Available at: at: https://www.nice.org.uk/guidance/cg143/chapter/Introduction [Accessed October 2021].
10. Ataga KI, et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439.

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