Vamorolone

AGAMREE

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Pronunciation: va-MOR-oe-loen (a-GAM-ree)

Vamorolone ballicule: receptor binding, kinetics and half-life diagram — Mechanism of action: Vamorolone ballicule

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.

Vamorolone molecular structure (skeletal formula) — Molecular structure: Vamorolone

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

Available strengths

susp 40 mg/mL

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

Cost

Vamorolone (AGAMREE)

Strength		#/mo	generic $/unit	CostPlus
40 mg/mL	susp	per unit		not offered

NADAC is a voluntary CMS survey of what retail community pharmacies pay for the drugs they stock. Vamorolone is dispensed outside retail pharmacies, so it rarely appears on surveyed invoices.

NO BOXED WARNINGS

Principal riskStill a steroid — adrenal suppression occurs

Classes and tags

Clinical profile

Therapeutic safety burden3 / 4

Overdose danger1 / 4

Weight-gain liability2 / 4

Sedation liability0 / 4

QT / Torsades0 / 4

Direct muscarinic antagonism0 / 4

Embryofetal risk2 / 4

Legacy pregnancy categoryN/A (post-2015 labeling)

Defining liabilityAdrenal suppression, Cushingoid weight gain and infection risk

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