Vamorolone

Vamorolone is a corticosteroid that acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which vamorolone exerts its effect in patients with DMD is unknown.

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

• AGAMREE is contraindicated in patients with known hypersensitivity to vamorolone or to any of the inactive ingredients of AGAMREE. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy [see Warnings and Precautions ( 5.13 )].contraindicated

The recommended dosage 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. ( 2.2 ) In patients with mild to moderate hepatic impairment, the recommended dosage is 2 mg/kg taken orally once daily preferably with a meal, up to a maximum daily dosage of 100 mg for patients weighing more than 50 kg. ( 2.3 ) Decrease dosage gradually when administered for more than one week. ( 2.7 ) 2.1 Assessments Prior to First Dose of AGAMREE Administer all immunizations according to immunization guidelines prior to starting AGAMREE. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting AGAMREE [see Warnings and Precautions ( 5.8 )]. 2.2 Dosing Information 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. 2.3 Recommended Dosage for Hepatic Impairment The recommended dosage of AGAMREE in patients with mild (Child-Pugh A) to moderate (Child-Pugh B) hepatic impairment is 2 mg/kg taken orally once daily preferably with a meal, up to a maximum daily dosage of 100 mg for patients weighing more than 50 kg [see Use in Specific Populations ( 8.6 ) and Clinical Pharmacology ( 12.3 )] . Doses may be titrated down based on individual tolerability.

Alterations in Endocrine Function: Hypothalamic-pituitary-adrenal axis suppression, cushingoid features, and hyperglycemia can occur. Monitor patients for these conditions with chronic use of AGAMREE. ( 2.7 , 5.1 ) Immunosuppression and Increased Risk of Infection: Increased risk of new infections, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; signs and symptoms of infections may be masked. ( 5.2 ) Alterations in Cardiovascular/Renal Function: Monitor for elevated blood pressure and monitor sodium and potassium levels in patients chronically treated with AGAMREE. ( 5.3 ) Gastrointestinal Perforation: Increased risk in patients with certain GI disorders; signs and symptoms may be masked. ( 5.4 ) Behavioral and Mood Disturbances: May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis. ( 5.5 ) Effects on Bones: Monitor for decreases in bone mineral density with chronic use of AGAMREE. ( 5.6 ) Ophthalmic Effects: May include cataracts, infections, and glaucoma; monitor intraocular pressure in patients chronically treated with AGAMREE. ( 5.7 ) Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting AGAMREE. ( 5.8 ) 5.1 Alterations in Endocrine Function Corticosteroids, such as AGAMREE, can cause serious and life-threatening alterations in endocrine function, especially with chronic use.

The following serious adverse reactions are discussed in more detail in other sections: Alterations in Endocrine Function [see Warnings and Precautions ( 5.1 )] Immunosuppression and Increased Risk of Infection [see Warnings and Precautions ( 5.2 )] Alterations in Cardiovascular/Renal Function [see Warnings and Precautions ( 5.3 )] Gastrointestinal Perforation [see Warnings and Precautions ( 5.4 )] Behavioral and Mood Disturbances [see Warnings and Precautions ( 5.5 )] Effects on Bones [see Warnings and Precautions ( 5.6 )] Ophthalmic Effects [see Warnings and Precautions ( 5.7 )] Immunizations [see Warnings and Precautions ( 5.8 )] Effects on Growth and Development [see Warnings and Precautions ( 5.9 )] Myopathy [see Warnings and Precautions ( 5.10 )] Kaposi's Sarcoma [see Warnings and Precautions ( 5.11 )] Thromboembolic Events [see Warnings and Precautions ( 5.12 )] Anaphylaxis [see Warnings and Precautions ( 5.13 )] The most common adverse reactions (>10% for AGAMREE and greater than placebo) are cushingoid features, psychiatric disorders, vomiting, weight increased, and vitamin D deficiency. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals, Inc. at 1-844-347-3277 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

8.1 Pregnancy Risk Summary AGAMREE is indicated for use for the treatment of DMD, which is a disease of young male patients. However, corticosteroids in general should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism. There are no data on the use of AGAMREE during pregnancy. Adverse developmental outcomes, including orofacial clefts (cleft lip, with or without cleft palate) and intrauterine growth restriction, and decreased birth weight, have been reported with maternal use of corticosteroids during pregnancy. Some epidemiologic studies report an increased risk of orofacial clefts from about 1 per 1000 infants to 3 to 5 per 1000 infants; however, a risk for orofacial clefts has not been observed in all clinical studies. Intrauterine growth restriction and decreased birth weight appear to be dose-related; however, the underlying maternal condition may also contribute to these risks (see Clinical Considerations and Data ) . Animal reproduction studies have not been conducted with AGAMREE. Animal reproduction studies conducted with corticosteroids in pregnant mice, rats, hamsters, and rabbits using clinically relevant doses have shown an increased incidence of cleft palate.

Metabolism

The major route of elimination is by metabolism with subsequent excretion of metabolites into urine. The pharmacokinetics (PK) are linear and vamorolone exposure increases proportionally with either single (0.1 to 20 mg/kg) or multiple (0.25 to 20 mg/kg) doses.

Treatment of acute overdosage of vamorolone is by immediate supportive and symptomatic therapy. Gastric lavage or emesis can be considered.

• Oct 26, 2023NDANDA215239Catalyst Pharms

1. 1Weight Increased21815%
2. 2Product Dose Omission Issue926.4%
3. 3Vomiting775.4%
4. 4Asthenia644.5%
5. 5Increased Appetite563.9%
6. 6Pyrexia563.9%
7. 7Influenza523.6%
8. 8Fatigue503.5%
9. 9Diarrhoea493.4%
10. 10Abdominal Pain Upper483.4%
11. 11Headache473.3%
12. 12Weight Decreased473.3%
13. 13Abdominal Discomfort463.2%
14. 14Fall453.2%
15. 15Gastroenteritis Viral422.9%

The 10 most recently updated of 12 ClinicalTrials.gov registrations naming Vamorolone as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
  Active not recruiting · Phase 4 · Interventional · 80 enrolled · Santhera Pharmaceuticals
  NCT06713135updated 2026-04-23
• Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
  Recruiting · Observational · 250 enrolled · Catalyst Pharmaceuticals, Inc.
  NCT06564974updated 2026-02-27
• Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
  Available · Expanded access · Santhera Pharmaceuticals
  NCT03863119updated 2026-01-20
• Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics
  Completed · Phase 1 · Interventional · 18 enrolled · Santhera Pharmaceuticals
  NCT06689527updated 2025-12-15
• A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
  Completed · Phase 2 · Interventional · 54 enrolled · Santhera Pharmaceuticals
  NCT05185622updated 2025-10-24
• A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
  Completed · Phase 2 · Interventional · 46 enrolled · ReveraGen BioPharma, Inc.
  NCT05166109updated 2025-09-18
• Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects
  Completed · Phase 1 · Interventional · 30 enrolled · Santhera Pharmaceuticals
  NCT06649409updated 2025-09-11
• A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
  Completed · Phase 2 · Interventional · 121 enrolled · ReveraGen BioPharma, Inc.
  NCT03439670updated 2023-03-09
• Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
  Completed · Phase 2 · Interventional · 46 enrolled · ReveraGen BioPharma, Inc.
  NCT03038399updated 2021-05-20
• Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
  Withdrawn · Phase 1 · Phase 2 · Interventional · 0 enrolled · ReveraGen BioPharma, Inc.
  NCT04348890updated 2020-09-29

Frequently asked questions

How does Vamorolone work?

Vamorolone is a corticosteroid that acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which vamorolone exerts its effect in patients with DMD is unknown.

What is Vamorolone used for?

According to FDA labeling, Vamorolone carries indications including: AGAMREE is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. AGAMREE is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Vamorolone?

Vamorolone is classified as Glucocorticoids, Corticosteroid, Corticosteroid Hormone Receptor Agonists, Glucocorticoid Receptor Agonists, Mineralocorticoid Receptor Antagonists, Immunologic Activity Alteration.

What are the brand names for Vamorolone?

Vamorolone is marketed under brand names including Agamree.

What are the contraindications for Vamorolone?

Vamorolone labeling lists contraindications including: AGAMREE is contraindicated in patients with known hypersensitivity to vamorolone or to any of the inactive ingredients of AGAMREE. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy [see Warnings and Precautions ( 5.13 )].. Always consult the full prescribing information and a clinician.