General Information

Agamree (vamorolone) is a corticosteroid.

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

Dosing/Administration

Agamree is supplied as an oral suspension.

Administer all immunizations according to immunization guidelines prior to starting Agamre. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting Agamree.

The recommended dosage of Agamree is 6 mg/kg taken orally once daily preferably with a meal, up to a maximum daily dosage of 300 mg for patients weighing more than 50 kg.

Some patients may respond to a dose of 2 mg/kg daily. Doses may be titrated down to 2 mg/kg/day as needed, based on individual tolerability.

Mechanism of Action

Agamree (vamorolone) is a corticosteroid that acts through the glucocorticoid receptor to exert antiinflammatory and immunosuppressive effects. The precise mechanism by which vamorolone exerts its effect in patients with DMD is unknown.

Side Effects

Adverse effects associated with the use of Agamree may include, but are not limited to, the following:

• cushingoid features
• psychiatric disorders
• vomiting
• weight increased
• vitamin D deficiency

Clinical Trial Results

The FDA approval of Agamree was based on data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, Agamree was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard of care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior.

In the pivotal VISION-DMD study, Agamree met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo at 24 weeks of treatment and showed a good safety and tolerability profile.