Hundreds of people in England with a life-limiting blood disorder are set to benefit from a world-first gene-editing therapy after it was given the green light for NHS use.
Health bosses hailed the decision by the National Institute for Health and Care Excellence (Nice) as a “historic moment” for patients with the condition.
Exagamglogene autotemcel, or exa-cel, is sold as Casgevy and made by Vertex.
The treatment is the first to be licensed using gene-editing tool Crispr , which earned its inventors the Nobel Prize for chemistry in 2020.
It works by modifying the faulty gene in a patient’s bone marrow stem cells so the body produces functioning haemoglobin. The edited cells are then infused back into the patient.
Casgevy has been recommended by Nice to treat patients aged 12 and over with transfusion-dependent beta thalassemia (TDT).
People with the inherited condition rely on regular lifelong blood transfusions to survive.
This is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS
Amanda Pritchard, chief executive of NHS England
The only curative treatment currently available for people with TDT in the UK is a donor stem cell transplant.
Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found.
According to the Nice guidance, the list price for a course of Casgevy is £1.65 million.
However, it is available to the NHS at a discount due to a commercial agreement between Vertex and the health service.
Nice recommended that Casgevy is used as part of the Innovative Medicines Fund (IMF).
It said this will allow more data on its clinical and cost effectiveness to be collected.
The treatment will be funded immediately and be rolled out to up to 460 eligible patients.
Amanda Pritchard, NHS England chief executive, said: “This is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS.
“Ordinarily, patients experience painful side effects and undergo regular transfusions which severely impact their quality of life, but this therapy offers people a life free from that as well as the hope of living longer, which is truly amazing news.
“This is the latest in a series of revolutionary gene therapies to be secured by NHS England over the past five years, bringing significant benefit for patients – and thanks to funding through our Innovative Medicines Fund, this one-off therapy will be fast-tracked to patients who could benefit from the new lease of life it promises.”
Helen Knight, director of medicines evaluation at Nice, added: “Although there are some uncertainties in the evidence for its long-term benefits, the committee felt exa-cel could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions.
“Today’s final draft guidance also addresses our aim of reducing health inequalities associated with conditions like transfusion-dependent beta-thalassaemia.”
I constantly worry about the future and what blood transfusions would be like when I’m older and how I will manage my condition
Kirthana Balachandran, who has thalassaemia
TDT mainly affects people of Pakistani, Indian and Bangladeshi ethnic origin.
Kirthana Balachandran, 21, from West London, was diagnosed with thalassaemia when she was three months old.
The medical student said: “I constantly worry about the future and what blood transfusions would be like when I’m older and how I will manage my condition.
“With gene therapy, I would potentially have a cure, and not have to have my three-weekly transfusions.
“I wouldn’t have to worry about the side effects of blood transfusions and the reactions I could have, such as iron depositing in my heart and liver, and what it could do to me.”
Professor Bola Owolabi, director of the National Healthcare Inequalities Improvement Programme at NHS England, said: “We are committed to reducing healthcare inequalities by rolling out new and pioneering treatments on the NHS for conditions, such as thalassaemia, which disproportionally affects people from some minority ethnic backgrounds.”
Ludovic Fenaux, senior vice president at Vertex International, said: “Securing access to Casgevy is a historic moment for people living with transfusion-dependent beta thalassaemia who, for too long, have had limited options for this life-shortening disease.
“Through collaboration with NHS England and Nice, we have reached this landmark agreement that recognises the value a one-time treatment can provide to patients, their families and the healthcare system.”
Yasmin Sheikh, head of policy and public affairs at stem cell charity Anthony Nolan, welcomed the Nice decision.
She said: “We’re delighted with this historic decision to approve the UK’s first ever Crispr-based therapy.
“Casgevy offers an effective cure for transfusion-dependent beta thalassaemia, a debilitating condition that was previously incurable in patients who don’t have a stem cell donor.”
In March, draft guidance from Nice did not recommend Casgevy for widespread NHS use to treat severe sickle cell disease after the watchdog said it needed “further data on its effectiveness, as well as a commercial arrangement”.
Ms Sheikh added: “This groundbreaking therapy must also be funded for people with sickle cell, where it has huge promise and is desperately needed.
“The sickle cell community has been waiting for months for Nice and the manufacturer of Casgevy, Vertex, to come to an agreement over bringing this therapy to the NHS.
“We hope this approval for thalassaemia demonstrates a solution is possible, and urge Nice and Vertex to work together to deliver this treatment to patients with sickle cell as soon as possible.”
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