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Dissociative steroid Pregnancy: Indicated for DMD, which is a disease of young male patients; there are no data on use during pregnancy. Corticosteroids in general should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Adverse developmental outcomes — including orofacial clefts (cleft lip, with or without cleft palate), intrauterine growth restriction and decreased birth weight — have been reported with maternal corticosteroid use during pregnancy. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism.

Vamorolone

Brand names: Agamree

Vamorolone is a corticosteroid-like anti-inflammatory medicine used to treat Duchenne muscular dystrophy.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: 6 mg/kg taken orally once daily, up to a maximum daily dosage of 300 mg for patients weighing more than 50 kg
Route: Oral — oral suspension 40 mg/mL; shake well for about 30 seconds before administration and use only the oral syringe provided, dispensing directly into the mouth. Preferably taken with a meal
Frequency: Once daily, preferably with a meal
Max: 300 mg daily for patients weighing more than 50 kg
Indicated for the treatment of Duchenne muscular dystrophy (DMD), largely a disease of children and young adults — there is no geriatric experience. Some patients may respond to a dose of 2 mg/kg daily, and doses may be titrated down to 2 mg/kg/day as needed based on individual tolerability. Mild (Child-Pugh A) to moderate (Child-Pugh B) hepatic impairment: the recommended dosage is 2 mg/kg taken orally once daily, up to a maximum daily dosage of 100 mg for patients weighing more than 50 kg; doses may be titrated down based on individual tolerability. With strong CYP3A4 inhibitors: the recommended dosage is 4 mg/kg taken orally once daily, up to a maximum daily dosage of 200 mg for patients weighing more than 50 kg. Switching from corticosteroid treatment: patients can be switched from oral corticosteroid treatment (such as prednisone or deflazacort) without treatment interruption or a period of prior corticosteroid dosage reduction, to minimise the risk of adrenal insufficiency; patients switching after long-term oral corticosteroid treatment should start at 6 mg/kg/day. Discontinuation: the dosage must be decreased gradually if the drug has been administered for more than one week. Before the first dose administer all immunizations according to guidelines, giving live-attenuated or live vaccines at least 4 to 6 weeks before starting. Discard any unused oral suspension remaining after 3 months of first opening the bottle. Source note: no UK SPC posology was available in the fetched bundle — this draft is from US labelling and must be verified against the UK SPC.

Paediatric dose

Dose: 6 mg/kg
Route: Oral (oral suspension 40 mg/mL)
Frequency: Once daily, preferably with a meal
Max: 300 mg daily for patients weighing more than 50 kg
The recommended dosage of 6 mg/kg once daily applies across the licensed population; safety and effectiveness for the treatment of DMD have been established in patients 2 years of age and older and have not been established below the age of 2 years. Some patients may respond to a dose of 2 mg/kg daily, and doses may be titrated down to 2 mg/kg/day as needed based on individual tolerability. With strong CYP3A4 inhibitors the recommended dosage is 4 mg/kg once daily (maximum 200 mg daily for patients weighing more than 50 kg); with mild to moderate hepatic impairment it is 2 mg/kg once daily (maximum 100 mg daily for patients weighing more than 50 kg). Decrease the dosage gradually if given for more than one week. Source: US labelling — verify against the UK SPC.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Paediatric weight-based calculator

The recommended dosage of 6 mg/kg once daily applies across the licensed population; safety and effectiveness for the treatment of DMD have been established in patients 2 years of age and older and have not been established below the age of 2 years. Some patients may respond to a dose of 2 mg/kg daily, and doses may be titrated down to 2 mg/kg/day as needed based on individual tolerability. With strong CYP3A4 inhibitors the recommended dosage is 4 mg/kg once daily (maximum 200 mg daily for patients weighing more than 50 kg); with mild to moderate hepatic impairment it is 2 mg/kg once daily (maximum 100 mg daily for patients weighing more than 50 kg). Decrease the dosage gradually if given for more than one week. Source: US labelling — verify against the UK SPC.

Verify in a children's formulary

Contraindications

  • Known hypersensitivity to vamorolone or to any of the inactive ingredients — instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy

Side effects

  • Cushingoid features (most common, >10% and greater than placebo)
  • Psychiatric disorders — behavioural and mood disturbances may include euphoria, insomnia, mood swings, personality changes, severe depression and psychosis
  • Vomiting
  • Weight increased
  • Vitamin D deficiency; decreases in bone mineral density with chronic use
  • Hypothalamic-pituitary-adrenal axis suppression and hyperglycaemia; increased risk of infection with immunosuppression

Interactions

  • Strong CYP3A4 inhibitors (e.g. itraconazole) — increase vamorolone exposure; reduce the dosage to 4 mg/kg once daily, up to a maximum daily dosage of 200 mg for patients weighing more than 50 kg
  • Moderate or weak CYP3A4 inhibitors — no dosage adjustment required
  • Live or live-attenuated vaccines — do not administer to patients receiving immunosuppressive doses of corticosteroids; give live-attenuated or live vaccines at least 4 to 6 weeks before starting treatment

Clinical monograph

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.

Reference: NICE TA1029; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

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