NICE recommends first licenced treatment for AL amyloidosis

Eligible individuals in England and Wales can access the first licensed treatment for amyloid light-chain (AL) amyloidosis on the NHS. The National Institute for Health and Care Excellence (NICE) recommended DARZALEX^® (daratumumab) plus VELCADE^® (bortezomib), cyclophosphamide and dexamethasone (DVCd) as an option for people newly diagnosed with the systemic form of the disease. 

According to the Janssen Pharmaceutical Companies of Johnson & Johnson and NICE, DVCd is recommended for the rare bone marrow disorder, only if daratumumab is stopped after 24 cycles of treatment, or earlier if the condition progresses. This decision is also subject to a commercial agreement.

“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,” stated Dr Sophie Castell, Chief Executive, Myeloma UK. She emphasised this recommendation is significant because DVCd “has been available in Scotland and Northern Ireland since 2022”.

NICE’s positive decision, which is detailed in the final appraisal document, follows an appeal against NICE’s original guidance from November 2022.

A promising treatment for AL amyloidosis 

According to a 2018 paper published in Front Immunology, daratumumab is a human monoclonal antibody that targets the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells.

In NICE’s final appraisal document, the DVCd regime was described as an effective and generally well-tolerated treatment. The document highlighted that daratumumab in combination “increases the time until systemic AL amyloidosis gets worse compared with bortezomib plus cyclophosphamide and dexamethasone.”

Amanda Cunnington, Senior Director of Patient Access, Janssen-Cilag Limited, shared that NICE’s decision is important because to date, “there have been limited options for those suffering with the symptoms of this serious condition.”

The Janssen Pharmaceutical Companies of Johnson & Johnson noted that Janssen Biotech, Inc. and Genmab A/S made an agreement in August 2012, in which Janssen would be granted an exclusive license to develop, manufacture and commercialise daratumumab.