Around 5000 patients are likely to benefit from the drug over the next 3 years

Trsakaoe iv intravenous drug

The first patients with sickle cell disease (SCD) have been treated by NHS England with the ‘life-changing’ drug, crizanlizumab (Adakveo; Novartis).

Loury Mooruth, 62, who was among the earliest patients to receive the drug, said: ‘Whenever I thought about having this new drug it brought tears to my eyes. I am so excited and over the moon because it is literally life-changing for me and my family.’

Crizanlizumab was recommended by NICE in October 2021 under a managed access agreement in partnership with NHS England. Under the deal, around 5,000 people are likely to benefit from the drug over the next 3 years. The treatment will be available at 10 new clinics dedicated to treating SCD across England.

A boon for sickle cell patients

SCD is a debilitating inherited blood disorder characterised by chronic pain, frequent visits to A&E, and reduced quality of life. Nearly 15,000 individuals in the UK are estimated to be currently affected by the condition, with a higher prevalence among individuals of African or Afro-Caribbean origin. 

Vaso-occlusive crisis (VOC), commonly called sickle cell crisis, is a key symptom of SCD. The sickling of red blood cells can lead to increased adhesion among themselves and with other blood cells and vessel walls, thereby narrowing smaller blood vessels and inducing episodes of acute pain. VOC episodes can last for days and may sometimes result in organ damage and other long-term complications.

Crizanlizumab is a humanised monoclonal antibody indicated for the treatment of recurrent VOCs in SCD patients aged 16 years and older. It is administered as an intravenous infusion alone or in combination with hydroxyurea/hydroxycarbamide, and acts by binding to P-selectin, a protein that facilitates cell adhesion.

Kye Gbangbola MB, Chair of the Sickle Cell Society, said: ‘Sickle cell is an underserved and under-recognised condition, so it is great to see new treatments being made available after over 20 years. We hope that this will be the first of many new treatments being made available to improve the lives of those living with sickle cell.’

Dr Bola Owolabi, NHS Director of Health Inequalities and a GP in the Midlands, added: ‘It’s fantastic that our first NHS patients have been given this ground-breaking and historic new treatment for sickle cell disease—the first in over 2 decades. This revolutionary treatment will allow patients to have a better quality of life, reduce trips to A&E by almost half, and ultimately help to save lives.’

This article was originally published on Medscape, part of the Medscape Professional Network.

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