‘Life-Changing’ Treatment to Target Sickle Cell Disease

Up to 4,000 people with sickle cell disease in England could benefit from the drug Voxelotor if recommended by their clinician, which will offer an additional treatment option, following its approval by the National Institute for Health and Care Excellence (NICE).

Voxelotor (Oxbryta®) will be administered in the form of a tablet taken once a day for eligible patients and could help reduce the need for blood transfusions and lead to fewer hospital appointments.

It could benefit patients who have a history of severe reactions to blood, or who may not wish to have blood transfusions.

In England, there are around 17,000 people living with sickle cell disease – an inherited blood disorders, with 250 new cases a year. It is generally more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage.

In sickle cell disease, a gene mutation causes red blood cells to become irreversibly sickle shaped, which can lead to haemolytic anaemia – a blood condition that occurs when your red blood cells are destroyed faster than they are replaced.

Over a long period, the disorder can cause severe organ damage and intense pain if damaged red blood cells block vessels and restrict oxygen supply, which can also lead to strokes and sight loss.

NHS chief executive, Amanda Pritchard, said:

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.

“The NHS has worked hard to make this life-changing treatment available at a fair price for the taxpayer, as part of our wider drive to improve the quality and experience of care for sickle cell patients, and tackle the stigma and inequalities they have told me they face.

“By improving the quality of life and reducing the need for hospital care, this new treatment option also has the potential to free up doctors, nurses and other clinicians to better support other patients, so we are acting fast to get it to the frontline immediately through our Innovative Medicines Fund.”

Voxelotor will be used to treat sickle cell anaemia in people aged 12 and older after it was recommended for use on the NHS in England by NICE.

The NHS will fund the treatment via the Innovative Medicines Fund (IMF), meaning that eligible patients can receive fast-tracked access to the drug.

It is another treatment option for people with this rare condition, which has historically had very few available treatments and can be taken alongside the commonly used drug hydroxycarbamide, or on its own.

The drug was shown in clinical trials to improve anaemia compared to standard care and was also likely to reduce the need for repeated blood transfusions.

Clinical trials showed that more than half (51%) of people given Voxelotor had an increase in haemoglobin (a protein inside red blood cells that carries oxygen around the body) which could improve symptoms and quality of life. Trial data also showed three-quarters (74%) of people taking the drug saw a noticeable improvement in how they felt.

Professor Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, said: “We are proud to make this new treatment available on the NHS – it could help thousands of patients living with sickle cell disease across the country to have a higher quality of life and experience fewer side effects, and represents a significant step forward in addressing the healthcare inequalities experienced by some of our communities.

“It is vital that we continue to get new drugs into the hands of NHS clinicians to improve the lives of people living with sickle cell disorder, like Jennifer.”

Dr Jennifer O’Connor, who was diagnosed with sickle cell disease as a child and treated at Whittington Health NHS Trust, said: “My life was a constant treadmill of both acute and chronic pain, but in 2022, I accessed a trial of Voxelotor, through the Early Access Medicine Scheme and, within weeks, I felt a very positive impact.

Over the last two years, I felt increased energy, less fatigue, less pain, and no side effects. My pain medication has reduced significantly and there have been no acute episodes of sickle cell or pain crisis.

“My overall quality of life for the first time felt ‘normal,’ I am thriving, and my family has noticed the changes.”

Helen Knight, director of medicines evaluation at NICE, said: “This is great news for people with sickle cell disease, particularly given the health inequalities experienced by people with the condition.

“Throughout our appraisal of Voxelotor the appraisal committee has always considered that it has the potential to address the need for effective treatments for sickle cell disease, as well as address NICE’s aim of reducing health inequalities.”