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Scottish Medicines Consortium issues interim acceptance to Novartis’ Adakveo®▼ (crizanlizumab) for preventing recurrent vaso-occlusive crises, the first treatment for sickle cell disorder in over 20 years

Adakveo® (crizanlizumab) receives interim acceptance by the Scottish Medicines Consortium (SMC) for preventing recurrent sickle cell crises (vaso-occlusive crises, VOCs) in people aged 16 or older with sickle cell disorder (SCD)1

• Significant step forward in the commitment to health equity for a community that has been historically underserved2

• Vaso-occlusive crises (VOCs), commonly known as pain crises, disrupt patients’ lives physically, socially, and emotionally, and can increase risk of organ damage, costly hospitalisations and early death3

London, UK, 11 July, 2022 - Novartis UK has announced that the Scottish Medicines Consortium (SMC) has issued interim acceptance to Adakveo® (crizanlizumab) for use in NHS Scotland as a clinically and cost-effective treatment option for the prevention of recurrent vaso-occlusive crises (VOC) in sickle cell disorder (SCD) patients aged 16 years and older, as either an add-on therapy to hydroxyurea/hydroxycarbamide (HC/HU) or as monotherapy in patients for whom HC/HU is inappropriate or inadequate).1 Access to this innovative treatment not only helps support people with SCD, but also demonstrates a commitment by NHS Scotland 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-19.2

“People living with sickle cell disorder and dealing with the recurrent pain episodes find that every aspect of their daily life is disrupted. For some people, their condition even impacts their career and education, affecting their overall quality of life,” said John James OBE, Chief Executive, Sickle Cell Society. “For too long, the sickle cell community has gone without new innovations to help manage this challenging condition. We welcome this decision as it offers hope for people living with this complex, life-limiting and life-long condition in Scotland, as well as reducing associated costs to NHS Scotland.”

SCD is a genetic blood disorder in which a patient’s red blood cells change shape and become sticky, which can restrict blood flow through vessels distributing blood around the body.4 VOCs are the resulting recurrent and unpredictable episodes of pain which can affect any part of the body, but usually involve the limbs and back.5,6 People with this condition suffer an increased risk of high blood pressure, eyesight loss, and kidney and urinary

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problems, along with long-term damage to internal organs, such as kidneys, liver, spleen,

heart and lungs.4,7

The decision follows close consultation between SMC, NHS Scotland and Novartis, as well as

professional and patient/carer groups, through the Patient and Clinician Engagement (PACE)

process. It clears the way for eligible people living with SCD in Scotland to access

crizanlizumab. The SMC’s interim acceptance option is open to medicines with a conditional

marketing authorisation and is subject to ongoing evaluation and future reassessment.8

“Many people with sickle cell disease experience recurrent painful crises which can result in

hospital admission. These can cause chronic organ damage and in some circumstances can

be life threatening” commented Dr Louisa McIlwaine, haematology consultant Greater

Glasgow and Clyde. “Even if a crisis episode does not result in admission, it significantly

impacts the quality of life of the patients. Not all patients respond to the current treatment

options available, and so there is a significant unmet need in managing this disease. Today’s

decision provides a new treatment option for this group for the first time in over 20 years.”

“This announcement further demonstrates Novartis’ commitment to patients living with rare

disease, bringing forward an innovative treatment option to address high unmet needs for

eligible patients living with sickle cell disorder,” said Marie-Andrée Gamache, Country

President, Novartis Innovative Medicines UK and Ireland. “At Novartis UK, we are proud to be

an active partner within the sickle cell community and are dedicated to collaborating with the

NHS and the entire health ecosystem to identify and implement healthcare solutions for

patients most in need, no matter their background or location.”

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Last Updated: 12-Jul-2022