National Institute for Health and Care Excellence (NICE) recommends first licensed treatment for newly diagnosed systemic amyloid light-chain (AL) amyloidosis
- Daratumumab combination is recommended for use within the NHS for newly diagnosed systemic amyloid light-chain (AL) amyloidosis[1]
- Eligible individuals throughout England and Wales can now access the first licenced treatment for people living with this life-threatening disease1
- AL amyloidosis is a rare and incurable bone marrow disorder that affects up to 600 people in the UK annually, and can cause serious organ damage1,[2]
High Wycombe, 29 February 2024 – The Janssen Pharmaceutical Companies of Johnson & Johnson is pleased to announce that the National Institute for Health and Care Excellence (NICE) has recommended DARZALEX® (daratumumab) plus VELCADE® (bortezomib), cyclophosphamide and dexamethasone (DVCd) as an option for newly diagnosed systemic amyloid light-chain (AL) amyloidosis.1 DVCd is recommended subject to a commercial agreement, and only if daratumumab is stopped after 24 cycles of treatment, or earlier if the condition progresses.1 This positive decision follows an extensive appraisal process including an appeal against the original guidance from NICE in November 2022, and means that eligible individuals throughout England and Wales can now access the first licenced treatment for this disease.1,[3]
Amyloid light-chain (AL) amyloidosis is a rare and incurable bone marrow disorder that affects up to 600 people in the UK annually, and can cause life-threatening organ damage.1,2 The condition can seriously affect an individual’s physical and mental health, and those with the most severe forms of AL amyloidosis can present with heart and renal failure.1 People with AL amyloidosis face considerable unmet needs due to the poor outcomes often associated with the current standard of care, which includes chemotherapy and a stem cell or bone marrow transplant.1
“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 treatment combination. 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,” said Dr. Sophie Castell, Chief Executive, Myeloma UK.* “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 treatment – a treatment which has been available in Scotland and Northern Ireland since 2022. It's disappointing that it took so long to get to this point. We will continue to push for the system to work faster and deliver for those who need it most.”
The DVCd regimen is an effective and generally well-tolerated treatment that increases the time until systemic AL amyloidosis worsens compared to bortezomib, cyclophosphamide and dexamethasone alone.1 Before the NICE acceptance of DVCd, there were no licensed treatment options available for people with the condition in England and Wales, although DVCd has been available on the NHS in Scotland and Northern Ireland since 2022.[4],[5]
“We are so pleased to have reached this outcome for those with AL amyloidosis. To date, there have been limited options for those suffering with the symptoms of this serious condition,” said Amanda Cunnington, Senior Director of Patient Access, Janssen-Cilag Limited. “This decision means that people in England and Wales can finally access the first licensed treatment for AL amyloidosis on the NHS. The commitment and drive of all stakeholders involved has been critical to achieving this outcome, and we hope that the progress made here shows what is needed for the UK healthcare system and the pharmaceutical industry to work together to address unmet needs for patients with rare diseases.”
*Myeloma UK has provided this quote voluntarily and has not been compensated for any media work.
[1] National Institute for Health and Care Excellence. Daratumumab in combination for untreated systemic amyloid light-chain amyloidosis. Final draft guidance. Available at https://www.nice.org.uk/guidance/gid-ta10656/documents/674. Last accessed February 2024.
[2] Myeloma UK. AL amyloidosis. Available at https://www.myeloma.org.uk/understanding-myeloma/related-conditions/al-amyloidosis. Last accessed February 2024.
[3] National Institute for Health and Care Excellence. Daratumumab in combination for untreated systemic amyloid light-chain amyloidosis. Final appraisal document. Available at https://www.nice.org.uk/guidance/gid-ta10656/documents/final-appraisal-determination-document. Last accessed February 2024.
[4] Scottish Medicines Consortium. Daratumumab (Darzalex). Available at https://www.scottishmedicines.org.uk/medicines-advice/daratumumab-darzalex-al-full-smc2447. Last accessed February 2024.
[5] Northern Ireland Formulary. Managed Entry Decisions. Available at https://niformulary.hscni.net/managed-entry/managed-entry-decisions/. Last accessed February 2024.
