Vamorolone

How it works

It binds the glucocorticoid receptor as a dissociative agonist, retaining anti-inflammatory activity while modulating mineralocorticoid receptor signalling, aiming to preserve muscle function with a potentially improved safety profile compared with conventional corticosteroids.

Prescribing in practice

• Like other corticosteroids it suppresses the adrenal axis, so it must not be stopped abruptly and patients need stress-dose cover and a treatment alert during illness, surgery or trauma.
• It carries class corticosteroid risks including infection susceptibility, behavioural and mood changes and effects on growth.
• Live vaccines should generally be avoided during treatment.

Monitoring

Monitor growth, weight and blood pressure, bone health, adrenal function and behavioural changes during long-term use.

Counselling the patient

• Never stop the medicine suddenly, and seek medical advice during illness or before surgery as extra steroid may be needed.
• Report signs of infection and any marked changes in mood or behaviour.

Evidence & guidelines

Its efficacy in Duchenne muscular dystrophy was demonstrated in the VISION-DMD randomised controlled trial.