vamorolone at a glance
Medicine class
Dissociative corticosteroid
Licensed use
Duchenne muscular dystrophy in patients aged 4 years and older
Form
Oral suspension measured with the supplied syringe
Food
Can be taken with or without food; avoid grapefruit
What is vamorolone used for?
UK-licensed use:
Treatment of Duchenne muscular dystrophy in patients aged 4 years and older.
How does vamorolone work?
Vamorolone selectively binds glucocorticoid receptors and reduces inflammatory gene signalling. It also blocks mineralocorticoid-receptor activation. Its precise therapeutic mechanism in Duchenne muscular dystrophy is not fully understood.
vamorolone preparations and strengths
Oral suspension
Route: Oral
Strengths: 40 mg/mL
How to take vamorolone
Use vamorolone exactly as prescribed by the specialist.
Shake the bottle well before measuring.
Use only the supplied oral syringe and give the measured amount slowly into the mouth while the patient is upright.
It may be taken with or without food. Avoid grapefruit and grapefruit juice.
After use, separate the syringe parts, rinse under cold running water and leave to air-dry.
It can be given through an enteral feeding tube without dilution. Do not mix it with feed; follow the tube-flushing instructions.
vamorolone side effects
Common or expected effects
- Rounded or swollen facial appearance
- Increased appetite or weight
- Irritability
- Vomiting
- Abdominal pain or diarrhoea
- Headache
Get urgent medical advice
- Possible adrenal crisis, including severe weakness, vomiting, dizziness, confusion or profound tiredness, needs urgent assessment.
- Seek prompt assessment for a severe or worsening infection, as corticosteroid effects can make infections harder to recognise.
vamorolone in pregnancy
There are no human pregnancy data for vamorolone, and animal studies found reproductive toxicity. It should not be used during pregnancy unless the prescriber decides treatment is clinically necessary. Effective contraception is required for people who could become pregnant.
vamorolone while breastfeeding
It is unknown whether vamorolone or its metabolites enter human milk, and risk to a breastfed infant cannot be excluded. The product information advises stopping breastfeeding during treatment; discuss this with the specialist team.
vamorolone interactions
The specialist and pharmacist should review prescription, over-the-counter and herbal products before treatment.
Strong CYP3A4 inhibitors, including clarithromycin, telithromycin, itraconazole and voriconazole
These can increase vamorolone exposure; specialist review and treatment adjustment may be needed.
Grapefruit or grapefruit juice
May increase vamorolone exposure and should be avoided.
Strong CYP3A4 or PXR inducers, including carbamazepine, phenytoin, rifampicin and St John’s wort
These may lower vamorolone concentrations and reduce its effect; an alternative may be required.
Common questions about vamorolone
Answers are fully visible for fast scanning and source review.
Is vamorolone a steroid?
Yes. It is a dissociative corticosteroid with anti-inflammatory effects through the glucocorticoid receptor.
Can vamorolone be stopped suddenly?
No. Sudden reduction or withdrawal can cause adrenal crisis, so changes must be directed by the specialist team.
Can live vaccines be given with vamorolone?
No. Live and live-attenuated vaccines are contraindicated during treatment and within the six weeks before it starts.
How should opened suspension be stored?
Keep the opened bottle upright in a refrigerator and discard unused suspension after three months.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.