Vamorolone
AGAMREE
What it isDissociative glucocorticoid receptor modulator; Duchenne muscular dystrophy.
Why this oneIt preserves muscle benefit with less growth suppression and bone loss than prednisone.
What limits itAdrenal suppression still occurs; it is a steroid. Cushingoid features and behavioral change.
FDA label
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| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Duchenne muscular dystrophy (DMD) | Adults & peds >=2 y | 6 mg/kg PO QD, preferably with a meal (may down-titrate to 2 mg/kg/day per tolerability) | 6 mg/kg QD | 300 mg/day (>50 kg) |
Renal Undefined
Hepatic Mild-moderate (Child-Pugh A/B): 2 mg/kg QD, max 100 mg/day (>50 kg); severe: no recommendation (no experience)
With strong CYP3A4 inhibitors: 4 mg/kg QD, max 200 mg/day (>50 kg)
Instructions
- Shake oral suspension ~30 s; use only the provided oral syringe, dispense directly into mouth
- Discard unused suspension 3 months after first opening bottle
- Taper gradually if used >1 week — do not stop abruptly (adrenal insufficiency risk)
- Switch from oral corticosteroids (prednisone/deflazacort) without interruption or prior taper; start 6 mg/kg/day after long-term corticosteroids
- Give all immunizations per guidelines before starting; live/live-attenuated vaccines >=4-6 weeks before starting
Cautions
- Endocrine: HPA-axis suppression with adrenal insufficiency on withdrawal (taper; stress-dose steroids may be needed), Cushing's syndrome, hyperglycemia — monitor glucose
- Immunosuppression and increased infection risk (incl. TB reactivation, severe varicella/measles); may mask signs of infection
- Cardiovascular/renal: monitor BP, sodium, potassium with chronic use
- GI perforation risk in certain GI disorders; signs may be masked
- Behavioral/mood disturbances (euphoria, insomnia, mood swings, depression, psychosis)
- Decreased bone mineral density — monitor with chronic use
- Ophthalmic: cataracts, infections, glaucoma — monitor intraocular pressure with chronic use
- Do not give live/live-attenuated vaccines during immunosuppressive doses
- Effects on growth and development; thromboembolic events; Kaposi's sarcoma; anaphylaxis (see label)
Adverse reactions
- Cushingoid features (29% at 6 mg/kg)
- Psychiatric disorders (21%)
- Vomiting (14%)
- Weight increased (11%)
- Vitamin D deficiency (11%)
Pregnancy Use only if benefit justifies fetal risk; corticosteroids associated with orofacial clefts, growth restriction, decreased birth weight; observe newborn for hypoadrenalism
Lactation No vamorolone data; systemic corticosteroids appear in human milk and could suppress growth — weigh benefit vs risk
NO BOXED WARNINGS
Principal riskStill a steroid — adrenal suppression occurs
Classes and tags
Clinical profile
Mechanism of action: Vamorolone ballicule
