Here’s What We Know About The New Drug For Sickle Cell Disease

There’s good news ahead for those who live with sickle cell disease. For the first time in 20 years, there’s a new sickle cell treatment that will keep thousands of people out of hospital over the next three years, NHS England has said.

Sickle cell disease, an incurable disease that affects 15,000 people in the UK, is caused by a gene that affects how red blood cells develop. Symptoms begin showing from early childhood and while the disease is lifelong and requires medication, many patients lead relatively normal lives.

However, they can suffer regular and painful episodes, caused by sickle-shaped red blood cells blocking the small blood vessels. This new drug, which is called crizanlizumab, is designed to reduce those episodes.

The drug is injected into a vein and can be taken by itself or alongside standard sickle cell treatment and regular blood transfusions. During the medical trial, patients taking crizanlizumab had a sickle-cell crisis 1.6 times a year on average, where normally they might experience one three times a year.

The National Institute for Health and Care Excellence (NICE) said the hope of reducing health inequalities for Black people, who are predominantly affected by sickle cell and often have poorer health to start with, made the drug worth recommending.

Nice called it “an innovative treatment”, while NHS chief executive Amanda Pritchard hailed it as an “historic moment” for those with sickle cell disease.

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