New hope for sickle cell disease as first new drug in 20 years is approved

- Advertisement -

A “revolutionary” life-changing drug treatment for sickle cell disease is to be made available on the NHS – the first new treatment for the disease in 20 years.

- Advertisement -

It will initially be made available to only about 300 patients, but will be increased to 450 in future years, following a decision by the National Institute for Health and Care Excellence (Nice), which recommended the drug be available on the NHS .

Sickle cell disease – which is especially common in people with African or Caribbean backgrounds – is a serious and lifelong health condition that causes severe pain and organ failure that often requires hospital admission.


The new drug, known as crizanlizumab, will be delivered by a transfusion drip and works by binding to a protein in blood cells to prevent restriction of blood and oxygen supply.

The NHS has said it could help 5,000 people over the next three years. It is hoped that the drug will reduce the number of sickle cell patients going to A&E by two-fifths.

- Advertisement -

People above 16 years of age who suffer from Multiple Sickle Cell Crisis every year will be eligible for the treatment.

Neiss said he cannot yet recommend the drug for routine use because of the high uncertainty about the long-term effectiveness of the treatment and the costs associated with it. The agreement with the NHS will allow patients to access the drug while additional data is collected through clinical trials.

The 28-year-old victim Adiola Omoyle, a resident of London, told Granthshala From the hospital: “It’s a catch-22 situation. It is good that at least some people will have additional treatment. But it is a pity that it has been 20 years since a new treatment drug for sickle cell and now only a small number of people will benefit from it. More needs to be done for the benefit of all.

“When you look at the disparities between other diseases like cancer, there is more research than sickle cell that mainly affects black people. Lack of awareness about sickle cell, lack of funding and resources and Issues of poor patient care need to be addressed.”

Laurel Brument, 57, of London, was diagnosed with sickle cell disease at the age of three.

Children’s author told Granthshala: “The fact that this is something new for sickle cell sufferers and will benefit someone who really needs it is positive. There are a lot of people with sickle cell disease out there who want access to this treatment. I wonder how the doctors will decide who to give it to when so many people have the condition?

Her symptoms were managed for 10 years with another drug called hydroxycarbamide – before which she suffered from persistent distress with pain described as “out of this world.” He said he hopes the new drug will bring relief to those who need it.

“If you had given me a drug that could have stopped it, and could have given me a better quality of life, I would have taken it,” she said.

Hayley King, founder of Sienna Smile, a charity that supports and educates families about sickle cell anemia in the UK, said: “We are extremely excited and relieved that a new treatment has finally been developed and for those affected. Will be available on the NHS.In the UK by sickle cell.

“Seeing developments in treatments is extremely reassuring. Sickle cell is the fastest growing genetic condition in the world and it is long overdue. We look forward to seeing other treatments that have already been approved by the FDA [United States Food and Drug Administration] Will also be approved by Nice and will be available on the NHS as soon as possible.

“It will give people options and opportunities to reduce the complications and symptoms they experience and life challenges many people with sickle cell face.”

NHS England chief executive Amanda Pritchard said: “This is a historic moment for people with sickle cell disease who will be given their first new treatment in two decades.

“This revolutionary treatment will help save lives, allow patients a better quality of life and cut A&E visits by almost half.

“The NHS has agreed a deal for this drug, so we are able to provide the latest and best possible treatment for patients at an affordable cost to taxpayers.”

Kai Gabangbola, president of the Sickle Cell Society, said: “A new treatment brings new hope to people living with sickle cell disorder, the world’s most common genetic blood condition.

“SCD is a medical emergency, it causes excruciating pain, this new treatment will reduce the number of excruciating pain episodes we have to endure.

“The hope is an improvement in the quality of life for many people living with the condition and their families.”


Credit: /

- Advertisement -
Mail Us For  DMCA / Credit  Notice

Recent Articles

Stay on top - Get the daily news in your inbox

Related Stories