‘Life-changing’ drug for victims of sickle-cell disease given go-ahead

A WONDER drug could change the lives of around 4,000 people with sickle-cell disease as a new treatment was given the green light for use in the NHS.

Health leaders said voxelotor, also known as oxbryta and made by Pfizer, could be “life-changing” and would significantly improve the quality of life for patients with the condition.

It was initially rejected for widespread NHS use but the National Institute for Health and Care Excellence has approved it in final draft guidance after a reduction in its price.

The Sickle Cell Society welcomed the news, saying the drug had “transformed” the lives of people with the condition.

John James, chief executive of the Sickle Cell Society, said: “Today’s approval marks a significant milestone in sickle-cell care, addressing decades of limited access to diseasemodifying treatments.

“Users of voxelotor have shared with us remarkable stories of improved quality of life, reduced pain and substantial increases in energy levels.

"This has allowed them to work, connect with friends and family, improve their physical and mental health and generally achieve a quality of life the rest of us often take for granted.

“We have seen decades of underinvestment in better care and safe effective treatments for sickle cell.”

People with sickle-cell disease produce unusually shaped red blood cells which can cause problems because the blood cells do not survive as long as healthy blood cells and can block blood vessels.

The drug works by helping haemoglobin – the protein in red blood cells – hold on to more oxygen and prevents red blood cells from becoming misshapen.

The drug, taken as a once-a-day tablet, will be available straight away. Amanda Pritchard, chief executive of NHS England, said the service had “worked hard to make this life-changing treatment available”, adding: “Sickle-cell disease can have a huge impact on people’s lives and this is one of the biggest breakthroughs in treatment in recent decades so I know how much this announcement will mean for thousands of people across the country.”

Susan Rienow, president of Pfizer UK, said: “We have remained committed to ensuring this treatment would reach eligible patients and we fully recognise the significance of this milestone for the sickle cell community.”

Health minister Andrew Stephenson said: “This is hugely welcome news that will help tackle long-standing health inequalities.”

There are around 17,500 UK sufferers and the condition is more common among people with an African or Caribbean background.