Stem cell charity Anthony Nolan welcomes the decision to make Casgevy (exa-cel) available to patients with the serious blood disorder thalassaemia, and calls for NICE to urgently approve the treatment for sickle cell patients who may also benefit.
Exa-cel, also known by the brand name Casgevy, is the first CRISPR-Cas9 therapy to be approved for use by the NHS. This type of gene therapy involves modifying a patient’s own stem cells to treat the underlying genetic causes of disease.
Today, NICE has issued final draft guidance recommending exa-cel should be funded for patients between the ages of 12 and 35 with transfusion-dependent beta thalassaemia (TDT) – an inherited condition that affects red blood cells, which are used to carry oxygen around the body.
People with TDT require regular blood transfusions, causing a build-up of iron in the body and serious complications. TDT shortens life expectancy and has many negative impacts on the quality of life of those affected.
Prior to this decision, the only curative treatment available to people with beta-thalassaemia was a donor stem cell transplant, however not all patients have a suitable donor available. Exa-cel can now provide a ‘functional cure’ for people unable to have a stem cell transplant by introducing healthy red blood cells into eligible TDT patients.
Unlike a stem cell transplant, which uses cells from a healthy donor, exa-cel involves genetically editing the patient’s own cells which are re-infused into them .
The therapy will initially be funded for three years through the Innovative Medicines Fund, a decision will then be made about longer-term funding Exa-cel will be available to patients living in England and Wales and will be delivered by a small number of specialist centres. NICE is also considering whether to recommend exa-cel for the treatment of patients with sickle cell, another blood disorder that also affects red blood cells.
Earlier this year NICE issued a draft decision not to recommend exa-cel for sickle cell, citing uncertainties in measuring the impact that severe sickle cell has on the quality of life of people affected. Anthony Nolan has campaigned with the Sickle Cell Society for this decision to be urgently reversed to ensure patients with sickle cell can have access to this potentially life-changing therapy.
Yasmin Sheikh, Head of Policy and Public Affairs said: "We’re delighted with this historic decision to fund 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.
“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.”
Sonal, who had Thalassaemia, said: “I was told as a child that I needed a stem cell transplant to cure my Thalassaemia, but unfortunately there was no match for me on the register or within my family. The doctors advised my parents to try for another baby - there would be no guarantee that the baby would be a match but there was a chance. My sister was born and luckily she was a match, so my transplant was able to go ahead when I was 5 years old.
“I’m so happy to see that exa-cel has now been approved for people like me. I can’t imagine how stressful it must have been for my parents to experience the pressure and anxiety of having another baby to help their sick child. Now, patients like me who have no match on the stem cell register or in their family will have a second chance at life."
Funmi Dasaolu, who has sickle cell, said: "Sickle cell disease has a profound impact on all aspects of my life. I can't remember a day when I haven't experienced pain and/or fatigue.
"It’s great news Exa-cel has been approved today for use in Thalassaemia; it offers much needed hope and a chance at a normal life. As a sickle cell community, we celebrate this milestone as we understand the daily struggles of living with a chronic, debilitating and distressing condition. We hope and eagerly anticipate Exa-cel will also be a potentially curative option for those who have sickle cell but do not have a stem cell donor in the very near future."
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For support related to thalassemia, please visit UKTS
For support related to sickle cell, please visit Sickle Cell Society