UK Adds New Sickle Cell Therapy Adakveo to National Health Service

Yedida Y Bogachkov PhD avatar

by Yedida Y Bogachkov PhD |

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A new treatment, called Adakveo (crizanlizumab), is now available to treat sickle cell disease (SCD) patients — the first time in decades that a new therapy for the disease has been made accessible — through the United Kingdom’s National Health Service (NHS), according to the U.K. Sickle Cell Society.

Adakveo will be available in England and Wales to eligible patients, 16 years and older, to help prevent recurrent sickle cell crises (pain episodes).

“This new treatment is long overdue, being the first licensed treatment for sickle cell in the UK in nearly thirty years, which illustrates how underserved sickle cell has been over the decades,” John James, Sickle Cell Society’s chief executive, said in a society press release.

Adakveo, developed by Novartis, is a monoclonal antibody that binds to P-selectin, a protein present on the surface of endothelial cells (the cells that line the inner walls of blood vessels) and in platelets (blood cells that are involved in clotting).

In SCD, P-selectin contributes to the adhesion of sickle-shaped red blood cells to blood vessels, thereby preventing proper blood flow through smaller vessels. This ultimately causes inflammation and leads to vaso-occlusive pain crises.

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During these crises, SCD patients are often admitted to the hospital and given strong pain medications, such as opioids. Some patients require regular blood transfusions to help reduce the number of crises.

Adakveo inhibits P-selectin, thus helping to reduce the frequency of vaso-occlusive crisis and increasing the time to first crisis occurrence.

“Sickle cell crises are extremely painful and disruptive to daily life, so it is very positive that a new treatment which can help reduce the number of crises for people living with sickle cell is being made available and funded by the NHS,” James said.

The new therapy can be given as an add-on treatment to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. HU/HC is often used to decrease the number of vaso-occlusive crises and works by increasing fetal hemoglobin; however, the treatment can have multiple side effects, including an increased risk of serious infections or bleeding.

Adakveo will be available on the NHS following a recommendation by the U.K. National Institute for Health and Care Excellence.

“We hope that this will be the first of many new treatments made accessible to improve the lives of those living with sickle cell, as well as enable sickle cell patients to have a wider choice of treatments,” James said.

An estimated 15,000 people in the U.K. have SCD.