Victory as first treatment for AL amyloidosis approved after NICE U-turn

Patients with AL amyloidosis have claimed a hard-fought victory after the very first treatment for the incurable disease was approved on the NHS, following a U-turn by NICE.

Up to 600 people each year will now benefit from the life-extending treatment, known as DaraCyBorD.

This is a huge step forward for people affected by AL amyloidosis, who currently rely on treatments designed to target other conditions.

It comes over a year after DaraCyBorD was initially rejected for newly-diagnosed patients in England and Wales by drug-approval body, NICE, on the grounds it was not cost-effective.

Myeloma UK immediately appealed the decision and launched the #UnlockDaraCyBorD campaign.

The first treatment of its kind, DaraCyBorD has been shown to be effective in clinical trials with patients five times more likely to see their disease stopped in its tracks.

59% of patients who received DaraCyBorD also had no sign of AL amyloidosis 20 months after treatment and longer remission times.

The treatment combines daratumumab (Darzalex®) with cyclophosphamide, bortezomib (Velcade®) and dexamethasone.

All four drugs are already individually available on the NHS.

Dr Sophie Castell, Chief Executive at blood cancer charity Myeloma UK, thanked patients, clinicians and all those who backed the #UnlockDaraCyBorD campaign and fought to make the new treatment available on the NHS. She said:

“This is tremendous news and means that, for the very first time, people who have AL amyloidosis will be able to gain access to this bespoke treatment. I want to extend my heartfelt thanks to everyone who has fought alongside us. Every person who has written to their MP, shared their story and helped us make the case to NICE about this significant unmet need and the huge impact it will have.

“This new drug combination is a game-changer and we know it can have a significant impact on people’s quality of life and remission times.

“Although we’re immensely proud of what we’ve achieved together, we are mindful that, over the last year, while we pushed for a U-turn on NICE’s decision, hundreds of newly-diagnosed patients missed out on this life-changing treatment – a treatment which has been available in Scotland since 2022. It’s disappointing that it took so long to get to this point.

“Patients deserve to receive the best, most effective treatments, no matter where they live. We will continue to push for the system to work faster and deliver for those who need it most.”

Between 500 and 600 people are diagnosed with AL amyloidosis in the UK every year.

Myeloma UK appealed against NICE’s decision to reject DaraCyborD back in December 2022. During the appeal process, the charity argued that NICE had acted unfairly – by failing to invite an AL amyloidosis expert or a haematologist to participate in the review process – and that decisions regarding treatment benefits and cost-effectiveness were unreasonable in light of the evidence submitted.

An independent panel eventually found against NICE on all counts.

14 months on, NICE has now reversed its initial decision and agreed to roll out the treatment on the NHS in England and Wales.

Professor Graham Jackson, Chief Clinical and Scientific Advisor at blood cancer charity Myeloma UK, said:

“This announcement has been long awaited and Myeloma UK have fought very hard to really make sure this often-ignored group of patients finally have a specific and active treatment that has been developed especially for their needs. DaraCyBorD has been shown to significantly improve response and resulted in marked improvements in heart and kidney function where those organs have been impacted by the diagnosis of AL amyloidosis. The continuous nature of this treatment should allow long-term control of the underlying disease and long-term improvements in function and quality of life.”

Michael Jameson, from Walsall, was just 41 when he was diagnosed with both AL amyloidosis and myeloma back in December 2021. He was initially given three months to live.

Thankfully, the father-of-two was able to receive DaraCyBorD through private healthcare. Two and a half years on, Michael has had a complete response to treatment.

“If I hadn’t received DaraCyBorD, three months later I might not have been around. When I was diagnosed my consultant asked me if I had private healthcare and I was lucky enough to be able to answer yes. I can’t imagine what it would have been like for those people who didn’t have private healthcare and those who perhaps haven’t survived as a result.”

“It’s great news it’s finally been approved on the NHS so that anybody in a similar situation to me, who doesn’t have that private healthcare to fall back on, has an approved treatment. And not just any treatment but probably one of the best treatments available for AL amyloidosis.”

Ross Cunliffe, a father-of-two from Woburn, Bedfordshire, was diagnosed with both AL amyloidosis and myeloma back in late 2013. He was 54 years old. Without DaraCyBorD, he faced a prognosis of just seven months.

“There was a strong likelihood I would not survive seven months,” said the 65-year-old, who has since been diagnosed with stage four lung cancer. “I am aware of how lucky I am to get these drugs privately. The seven months became completely redundant thanks to the treatment. But I could just have easily been in a completely different situation where it didn’t happen. I’m convinced beyond any shadow of doubt that these drugs have made me live longer, enabled me to spend more time with my grandchildren and enjoy massive chunks of life.”

What does this decision mean for patients?

Newly-diagnosed AL amyloidosis patients in England and Wales can now receive DaraCyBorD as their initial treatment.

The phase 3 ANDROMEDA trial assessed the effectiveness of DaraCyBorD compared to CyBorD, the current treatment for newly-diagnosed AL amyloidosis patients. The trial showed that adding daratumumab to the combination increased response rates and progression free survival (the amount of time before the AL amyloidosis comes back or organ damage gets worse).

Access to this treatment will increase the response rates, remission times and life expectancy of AL amyloidosis patients.

When will the treatment be available to patients in England and Wales?

When a treatment is recommended it should be made available to patients through the NHS within 90 days of the publication of the final decision.

The final decision is published 15 working days after the public has been notified of the final decision following the standard notice period for any appeal requests.

What about patients in Scotland and Northern Ireland?

The treatment is already available on the NHS for patients in Scotland. The Scottish Medicines Consortium (SMC) approved the treatment for use on the NHS in 2022.

 The Department for Health in Northern Ireland decides which treatments to fund based on decisions from either NICE or SMC. Therefore, patients in Northern Ireland can access DaraCyBorD.

What is DaraCyBorD?

DaraCyBorD is a combination of four drugs:   

  • Daratumumab (Darzalex®)   
  • Cyclophosphamide   
  • Bortezomib (Velcade®)   
  • Dexamethasone   

Daratumumab (Darzalex®) belongs to a group of drugs known as monoclonal antibodies. It works innovatively by harnessing the body’s immune system.

Daratumumab is a synthetic antibody designed to target AL amyloidosis and myeloma cells by detecting a protein commonly found on the surface of AL amyloidosis and myeloma cells called CD38. When daratumumab finds and attaches to CD38, it makes the abnormal cell more visible to the immune system. It helps the patient’s immune system locate and kill abnormal cells.

Daratumumab has been used to treat myeloma in England and Wales since 2018. However, it was not approved for use in AL amyloidosis – until now.

If you have any questions or would like to speak to us for information, advice and support, please call 0800 980 3332 or email askthenurse@myeloma.org.uk.

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