Around 2600 people with untreated multiple myeloma and over 23,000 people with ulcerative colitis in England are set to benefit from new treatment options following final draft guidance from the National Institute for Health and Care Excellence (NICE).
The regulator recommended daratumumab (Darzalex, Janssen) with lenalidomide and dexamethasone as an option for adults with untreated multiple myeloma when an autologous stem cell transplant is unsuitable.
Around 6000 people are diagnosed with multiple myeloma every year in England, and an estimated 2600 people could be eligible for the newly recommended treatment, according to NICE.
Clinical evidence for daratumumab plus lenalidomide and dexamethasone compared with lenalidomide plus dexamethasone came from the MAIA trial — an ongoing, randomised, open-label, multicentre, phase 3 trial — which includes adults with previously untreated multiple myeloma who could not have an autologous stem cell transplant. At the October 2021 data cutoff, the three-drug combination reduced the risk of disease progression and death by 45% compared with lenalidomide plus dexamethasone, which is usually the first treatment option. Median progression-free survival was 61.9 months in the daratumumab plus lenalidomide and dexamethasone group compared with 34.4 months in the lenalidomide plus dexamethasone group.
The NICE appraisal committee concluded that the MAIA trial had shown that daratumumab plus lenalidomide and dexamethasone was a "clinically effective treatment", but that longer-term overall survival was uncertain.
Daratumumab is a synthetic monoclonal antibody designed to target myeloma cells by detecting the CD38 protein commonly found on their surface, and making the myeloma cells more visible to the immune system, helping this system to locate and kill myeloma cells.
According to Myeloma UK, the combination of daratumumab plus lenalidomide and dexamethasone is "one of the most effective treatments" for newly diagnosed patients who are older, frailer, and unsuitable for stem cell transplantation. Shelagh McKinlay, director of research and advocacy at the charity, described the triple drug combination as a "game changer" that would make a tremendous difference to patients' quality of life.
The charity said that daratumumab had proven to be a "real step change" for myeloma treatment, with the drug having been introduced "earlier and earlier" in patients' treatment pathways. "Approximately two-thirds of newly diagnosed myeloma patients are not eligible for a transplant, and now, at long last, they'll be able to benefit from a life-extending treatment that could give them precious years with their loved ones," Ms McKinlay said.
"Promising Step Forward"
NICE has also recommended mirikizumab (Omvoh, Eli Lilly) as an option for people with moderate to severely active ulcerative colitis, with an estimated 23,000 people set to benefit.
The regulator stated that the drug is recommended as an option for treating moderately to severely active ulcerative colitis in adults when conventional or biological treatment could not be tolerated, or the condition had not responded well enough or lost response to treatment, but only if a tumour necrosis factor (TNF)‐alpha inhibitor had not worked,
could not be tolerated, or was not suitable.
The company must also provide the drug to the NHS at a discount under the terms of a confidential agreement, NICE stressed.
TNF‐alpha inhibitors are the most-used biological treatments for moderately to severely active ulcerative colitis, but when these didn't work, or were not tolerated, people were usually offered vedolizumab or ustekinumab, explained NICE.
"Mirikizumab is another biological treatment that would be offered to the same population as these two treatments," NICE confirmed.
Clinical trial evidence had showed that mirikizumab was more effective than placebo for treating moderately to severely active ulcerative colitis. However, NICE pointed out that there were no clinical trials directly comparing mirikizumab with vedolizumab or ustekinumab. However, an indirect comparison suggested that "all three treatments are similarly effective". Moreover, NICE stressed that the costs of mirikizumab were similar or lower to those of vedolizumab and ustekinumab.
Sarah Sleet, chief executive of Crohn's & Colitis UK, told Medscape News UK, that over 500,000 people in the UK were living with Crohn's disease and ulcerative colitis, and pointed out that "existing medications may not work for some people, or indeed stop working for others".
The charity welcomed the decision by NICE to recommend mirikizumab, which Ms Sleet said expanded the treatment options for eligible people living with colitis and was a "promising step forward".
Guidance from NICE applies to England only, but directions are usually adopted in Wales.