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Vamorolone (Agamree; Santhera), a corticosteroid, has been recommended for the treatment of Duchenne muscular dystrophy (DMD) in people aged four years and over by the National Institute for Health and Care Excellence (NICE).
In final draft guidance published on 10 December 2024, NICE recommended the treatment as an alternative to other available corticosteroids used to reduce muscle inflammation and maintain muscle strength and function in people with DMD.
NICE said that the treatment would be available to around 1,700 people in England and it could offer “important benefits because of its potential to reduce adverse events” associated with currently available corticosteroids, which can include osteoporosis, weight gain and delayed puberty.
DMD is an incurable genetic condition that causes progressive muscle loss. It primarily affects boys and is usually diagnosed between the ages of three years and six years. Patients with the condition have decreased life expectancy, mostly living into their 20s and 30s.
Other corticosteroids used in the treatment of the condition include prednisone, prednisolone and deflazacort.
NICE previously concluded that more evidence was needed to establish the cost-effectiveness of vamorolone. However, in the final draft guidance, NICE said that new analysis presented by the manufacturer, along with an improved discount to the price of the treatment, meant that it could recommend it as a “cost-effective use of NHS resources”.
Commenting on the guidance, Rob Burley, director of care, campaigns and support at the charity Muscular Dystrophy UK, said: “It’s great news that NICE has recommended vamorolone for people aged four [years] and over with DMD in England, giving individuals and families an alternative treatment option.
“We’re proud to have played a key role in the appraisal process, including nominating and supporting a patient expert to share her son’s experience of receiving the treatment through a clinical trial. Throughout the appraisal process, we heard first-hand about the dilemma that families face when it is recommended that their child starts taking corticosteroids due to the associated side effects, and the benefits an alternative choice would bring.”
Helen Knight, director of medicines evaluation at NICE, said: “The committee felt vamorolone could offer important benefits to patients and their families because of its potential to reduce adverse events associated with current steroid treatments.
“Given the significant burden this disease places on children with the condition and on their parents and carers, as well as the negative impact this can have on their quality of life, a treatment with the potential to reduce side effects associated with current treatments is to be welcomed.”
“We’re therefore delighted the company has been able to work with us to enable vamorolone to be made available to people with DMD,” she added.
In its statement, NICE said vamorolone is “one of several non-cancer treatments recommended by NICE following the introduction of the new severity modifier”.
“This allows NICE’s independent committees to give greater weight to severe conditions, including conditions such as DMD, that would not have qualified for additional weighting under the previous end of life criteria,” it added.
NICE introduced the severity modifier in February 2022, replacing the previous ‘end-of-life modifier’, to provide an additional weighting to the ‘value’ of some medicines that treat more severe conditions.
In November 2024, a report by the Office of Health Economics said the severity modifier may be blocking innovative treatments that the public would like to see benefit NHS patients.
