AGAMREE Oral Suspension – Treatment for Duchenne Muscular Dystrophy

AGAMREE is an oral corticosteroid suspension containing vamorolone, approved for treating Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. It helps manage this progressive muscle disease by modulating inflammation and immune responses.

AGAMREE is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

vamorolone

AGAMREE contains vamorolone, a corticosteroid that works by reducing inflammation and modulating the immune system. This helps slow muscle damage and improve muscle function in patients with Duchenne muscular dystrophy.

General Dosage

• 6 mg/kg orally once daily, preferably with a meal.
• Maximum dose: 300 mg/day for patients weighing over 50 kg.

Dosage Adjustments

• Some patients may respond to 2 mg/kg daily; doses may be adjusted downward based on tolerability, with as low as 2 mg/kg/day.
• In patients with mild to moderate hepatic impairment (Child-Pugh A or B), the recommended dose is 2 mg/kg once daily, up to 100 mg/day for those weighing more than 50 kg.
• When given with strong CYP3A4 inhibitors, reduce dose to 4 mg/kg once daily, up to 200 mg/day for patients over 50 kg; doses may be titrated downward based on tolerance.

Administration Instructions

• Shake oral suspension well for about 30 seconds before use.
• Use only the provided oral syringe to measure dose and administer directly into the mouth.
• Discard unused suspension 3 months after opening the bottle.

Switching from Other Corticosteroids

• Patients switching from oral corticosteroids (e.g., prednisone or deflazacort) can do so without interruption or prior dose tapering to minimize adrenal insufficiency risk.
• For those switching after long-term corticosteroid use, start AGAMREE at 6 mg/kg/day.

Discontinuation

• For treatments longer than one week, decrease AGAMREE dose gradually to prevent adrenal insufficiency.

AGAMREE is contraindicated in patients with known hypersensitivity to vamorolone or any of the inactive ingredients in the formulation.

Endocrine Effects

• Risk of hypothalamic-pituitary-adrenal (HPA) axis suppression and secondary adrenal insufficiency upon withdrawal; taper dose gradually.
• Symptoms of Cushing's syndrome with prolonged use (e.g., hypertension, truncal obesity, muscle weakness).
• Monitor blood glucose as hyperglycemia and diabetes may develop or worsen.
• Caution with altered thyroid function and risk of pheochromocytoma crisis.

Infection Risk

• Immunosuppression may increase risk of new, reactivated, or disseminated infections including bacterial, viral (e.g., varicella, measles), fungal, protozoan, or helminthic pathogens.
• Infections can be severe or fatal and may present with masked symptoms.
• Administer all immunizations according to guidelines prior to starting AGAMREE; avoid live or live-attenuated vaccines during treatment.
• Monitor closely for tuberculosis reactivation and hepatitis B reactivation; consider prophylaxis or antiviral therapy if indicated.
• Avoid use in systemic fungal infections unless necessary; withdraw or reduce dose if fungal infection develops.

Cardiovascular and Electrolyte Effects

• Monitor blood pressure and electrolytes (sodium, potassium) due to risks of hypertension, fluid retention, and hypokalemia.
• Use caution in patients with congestive heart failure, hypertension, or renal insufficiency.
• Potential association with myocardial infarction complications; use cautiously post-MI.

Gastrointestinal Concerns

• Increased risk of GI perforation in patients with conditions such as peptic ulcers or diverticulitis; symptoms may be masked.
• Avoid AGAMREE with impending GI perforation, abscess, or pyogenic infections.

Neuropsychiatric Effects

• Possible behavioral or mood disturbances including euphoria, insomnia, mood swings, depression, psychosis.
• Symptoms can appear within days to weeks and may improve with dose reduction or discontinuation.
• Caregivers should monitor for behavioral changes and seek medical attention for concerning symptoms.

Bone and Eye Effects

• Long-term use can reduce bone mineral density leading to osteoporosis and risk of fractures; monitor bone health.
• Risk of avascular necrosis and cataracts; monitor intraocular pressure if used over 6 weeks.
• Corticosteroids may increase risk of secondary ocular infections; not recommended in active ocular herpes simplex.

Other Warnings

• Use caution in patients predisposed to thromboembolism.
• Rare anaphylaxis and hypersensitivity reactions may occur.

Common and Noted Reactions

• Behavioral and mood changes including irritability, aggressive behavior, sleep disorders, especially in children.
• Signs of Cushing's syndrome with long-term use.
• Hyperglycemia or worsened diabetes.
• Immunosuppression-related infections.

Serious Adverse Events

• Anaphylaxis and hypersensitivity reactions.
• Adrenal insufficiency after abrupt withdrawal.
• Gastrointestinal perforation in predisposed patients.
• Pheochromocytoma crisis in susceptible individuals.
• Psychiatric symptoms including severe depression and psychosis.
• Bone mineral density reduction and avascular necrosis.
• Ophthalmic complications such as cataracts and glaucoma.

• Strong CYP3A4 inhibitors require dose reduction of AGAMREE to 4 mg/kg once daily, max 200 mg/day for patients >50 kg.
• Concomitant levothyroxine use: start AGAMREE before levothyroxine to minimize adrenal crisis risk.

Overdose may result in signs of corticosteroid excess. Management should be supportive and include gradual tapering of dose to avoid adrenal insufficiency.

Discard any unused AGAMREE oral suspension 3 months after opening. Shake well before use.