First treatment for sickle cell side-effect in 20 years approved

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC). 

The SMC issued interim acceptance for Novartis UK’s Adakveo for use in NHS Scotland as a treatment option for the prevention of recurrent vaso-occlusive crises (VOC) in SCD patients. Specifically, the acceptance is for Adakveo to be used either an add-on therapy to hydroxyurea/hydroxycarbamide (HC/HU) or as monotherapy in patients for whom HC/HU is inappropriate or inadequate. 

SCD is a genetic blood disorder in which patient’s red blood cells change shape and become sticky, which can restrict blood flow through vessels distributing blood around the body. VOCs are a common side effect in which a person with SCD experiences recurrent and unpredictable episodes of pain, usually in their limbs or back.  

People with this condition suffer an increased risk of high blood pressure, eyesight loss, and kidney and urinary problems, along with long-term damage to internal organs. 

Official comments  

“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.”  

“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,” added 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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