Adakveo Available in UK’s NHS for Patients 16 and Older

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Adakveo (crizanlizumab), by Novartis, is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older.

The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of painful episodes caused by blood vessel obstruction called vaso-occlusive crises (VOCs).

Adakveo is approved under a conditional marketing authorization that requires the company to provide more evidence about its safety and effectiveness. This means that to get access to the NHS-funded treatment, available as of Feb. 1, a managed access agreement was put in place to enable researchers to collect data from patients and determine whether they are benefiting from treatment.

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The agreement follows a recommendation from the National Institute for Health and Care Excellence (NICE) for the use of Adakveo as a therapeutic option for preventing painful crises. As new data arise, NICE will make a final recommendation about whether Adakveo should be funded for routine use in the NHS, which is anticipated to happen in 2025.

“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, chief executive at the Sickle Cell Society, said in a press release.

SCD is a genetic condition in which red blood cells acquire an unusual sickle-like shape, becoming stiffer and stickier. Because they have difficulty passing through blood vessels, they can block blood flow. This blockage can cause a painful crisis that requires pain killers to be taken at home or in a hospital.

“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.

Adakveo is a lab-made monoclonal antibody designed to bind to P-selectin, a protein found on the surface of the cells lining blood vessel walls. P-selectin helps other cells stick to blood vessels, which may contribute to painful SCD crises. By binding and blocking the action of P-selectin, Adakveo is expected to help prevent painful crises.

It can be used either in combination with hydroxyurea or on its own in patients for whom hydroxyurea causes too many side effects or does not work well enough.

It was announced in October that, for the first time in decades, a new treatment for SCD was going to be become available on the NHS. To be eligible to receive Adakveo, patients must have a confirmed diagnosis of SCD, be 16 or older, and have had two or more VOCs in the previous year.

“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,” said James.