AGAMREE VAMOROLONE Kit
🆔 Identity & classification
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🏷️ RxNorm drug class
This medicine belongs to the Corticosteroid class.
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🏭 Manufacturer & labeler
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🩺 Clinical
Vamorolone is used to treat Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly) in adults and children 2 years of age and older. Vamorolone is in a class of medications called corticosteroids. It works by reducing inflammation (swelling) and by changing the way the immune system works.
Read the full MedlinePlus article ↗Patient education
Supplement & herbal interactions
Some supplements/herbs that may interact with Vamorolone — tap one for details:
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💊 What it looks like
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🧪 Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
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IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.Inactive ingredient FAQ
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💲 Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per each | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | $101.44 | — |
| Medicare drug plans payPart D · Q2 2026 | $112.28 | — |
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🔁 Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Agamreethis 69616-0264-38 | Catalyst | 1 kit | — | — | FDA listed | — |
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⏳ Availability & generic status
We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.
Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.
🛈 What do these terms mean?
- Patent
- Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
- Substance patent
- Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
- Formulation (product) patent
- Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
- Method-of-use patent
- A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
- Skinny label
- A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
- Exclusivity
- FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
- Paragraph IV
- A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
- RLD / RS
- Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
- TE / AB rating
- FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
- LOE (loss of exclusivity)
- The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.
Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.
| Patent | Type | Use code | Expires |
|---|---|---|---|
| US 12201639 ↗ | Drug substance | U-3747 | Mar 17, 2040 |
| US 11690853 ↗ | Method of use | U-3747 | Mar 7, 2033 |
| US 11471471 ↗ | Method of use | U-3747 | Mar 17, 2040 |
| US 10857161 ↗ | Method of use | U-3747 | May 28, 2029 |
| US 8334279 ↗ | Method of use | U-3747 | May 28, 2029 |
| US 11833159 ↗ | Drug product | — | May 28, 2029 |
| US 11382922 ↗ | Drug substance | — | Jul 16, 2040 |
| Code | What it grants | Expires |
|---|---|---|
| NCE | New Chemical Entity (5-year) | Oct 26, 2028 |
| ODE-450 | Orphan Drug Exclusivity (7-year) | Oct 26, 2030 |
Is there a generic version of AGAMREE 40 MG/ML SUSPENSION?
The FDA approved a generic — why can’t I get it at my pharmacy yet?
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🗺️ Medicaid utilization & spend
📊 Medicare Part D spend CMS · PART D · 2026 (Q1)
🔬 Reported adverse events (FAERS)
Top reported reactions
Age at onset
Reporter sex
Serious outcomes
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📦 Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Status |
|---|---|---|---|
| 69616-0264-38 You're viewing this | 1 KIT in 1 CARTON (69616-264-38) * 100 mL in 1 BOTTLE, GLASS (69616-265-38) | 2024-02-01 | Active |
🧭 About this NDC listing & data coverage
Kit / multi-component package
This NDC identifies a kit — a package containing more than one component. Structured data (pricing, ingredients, equivalents) is often reported per component rather than for the kit NDC itself, which can make this page look thinner than the components' own pages.
What data is (and isn’t) available for this NDC — tap to expand
| NDC identity (package / product / labeler codes) | ✓ Available |
| Labeler | ✓ Available |
| Product & package description | ✓ Available |
| Marketing category & status | ✓ Available |
| Active ingredient / dosage form / route | ✓ Available |
| FDA label (SPL via DailyMed) | ✓ Available |
| Package photos | ✓ Available |
| Inactive ingredients (structured) | — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is available. |
| NADAC pharmacy acquisition price (CMS) | — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey. |
| Orange Book / therapeutic-equivalence data | ✓ Available |
| HCPCS J-code billing crosswalk | — Not published for this NDC Most self-administered / retail products have no J-code — that is normal. |
| Medicaid utilization (CMS SDUD) | ✓ Available |
Questions about this listing
Why is there no price listed?
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📄 Full prescribing information FDA SPL
🎯 Indications and Usage ▾
1 INDICATIONS AND USAGE 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. ( 1 )
⏱️ Dosage and Administration ▾
2 DOSAGE AND ADMINISTRATION 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 )
2.1Assessments 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 )].
2.2Dosing 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.
2.3Recommended 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.
2.4Important Preparation and Administration Instructions Shake AGAMREE oral suspension well for about 30 seconds before administration. Use only the oral syringe provided with the product. After withdrawing the appropriate dose into the oral syringe, dispense directly into the mouth. Discard any unused AGAMREE oral suspension remaining after 3 months of first opening the bottle.
2.5Switching from Corticosteroid Treatment to AGAMREE Patients can be switched from oral corticosteroid treatment (such as prednisone or deflazacort) to AGAMREE without treatment interruption or period of prior corticosteroid dosage reduction to minimize the risk for adrenal insufficiency. Patients switching after long-term treatment with oral corticosteroids should start AGAMREE at a dosage of 6 mg/kg/day.
2.6Dosage Modification for Use with Strong CYP3A4 Inhibitors The recommended dosage of AGAMREE when administered with strong CYP3A4 inhibitors is 4 mg/kg taken orally once daily preferably with a meal, up to a maximum daily dosage of 200 mg for patients weighing more than 50 kg [see Drug Interactions ( 7.1 ) and Clinical Pharmacology ( 12.3 )]. Doses may be titrated down based on individual tolerability.
2.7Discontinuation Dosage of AGAMREE must be decreased gradually if the drug has been administered for more than one week [see Warnings and Precautions ( 5.1 )].
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS Oral Suspension: 40 mg/mL orange flavored white to off-white suspension Oral Suspension: 40 mg/mL ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS 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 )]. Hypersensitivity to vamorolone or any of the inactive ingredients in AGAMREE ( 4 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS 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 )
5.1Alterations in Endocrine Function Corticosteroids, such as AGAMREE, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving AGAMREE for Cushing's syndrome, hyperglycemia, and adrenal insufficiency after AGAMREE withdrawal. In addition, patients with hypopituitarism, primary adrenal insufficiency or congenital adrenal hyperplasia, altered thyroid function, or pheochromocytoma may be at increased risk for adverse endocrine events.
Risk of Adrenal Insufficiency Following Withdrawal AGAMREE produces reversible hypothalamic-pituitary-adrenal (HPA) axis suppression, with the potential for the development of secondary adrenal insufficiency after withdrawal. Acute adrenal insufficiency can occur if AGAMREE is withdrawn abruptly, and could be fatal. The degree and duration of adrenocortical insufficiency produced is variable among patients and depends on the dose and duration of therapy.
The risk of adrenal insufficiency is reduced by gradually tapering the dose when withdrawing treatment. The insufficiency may persist, however, for months after discontinuation of prolonged therapy; therefore, in any situation of stress occurring during that period of discontinuation, supplementation with a systemic corticosteroid is recommended. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.
A steroid “withdrawal syndrome”, seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. This syndrome includes symptoms such as anorexia, nausea, vomiting, lethargy, headache, fever, joint pain, desquamation, myalgia, and/or weight loss. These effects are thought to be due to the sudden change in corticosteroid concentration rather than too low corticosteroid levels.
Cushing's Syndrome Cushing's syndrome (hypercortisolism) occurs with prolonged exposure to exogenous corticosteroids, including AGAMREE, and symptoms include hypertension, truncal obesity and thinning of the limbs, purple striae, facial rounding, facial plethora, muscle weakness, easy and frequent bruising with thin fragile skin, posterior neck fat deposition, osteopenia, acne, amenorrhea, hirsutism, and psychiatric abnormalities. Hyperglycemia Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term therapy to diabetes mellitus, and may reduce the effect of anti-diabetic drugs.
Monitor blood glucose at regular intervals in patients treated with AGAMREE. For patients…
🤒 Adverse Reactions ▾
6 ADVERSE REACTIONS 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.
6.1Clinical 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. Common Adverse Reactions in Clinical Studies Table 1 lists the adverse reactions that occurred in ≥ 5% of the patients treated with AGAMREE 6 mg/kg/day (N=28) or AGAMREE 2 mg/kg/day (N=30) and that occurred more frequently than in the patients who received placebo (N=29) in Study 1 [see Clinical Studies ( 14 )] , which was 24 weeks and included patients with DMD between the ages of 4 and 7 years.
Table 1: Adverse Reactions in Patients with DMD that Occurred in ≥ 5% of Patients Treated with AGAMREE and More Frequently than in Patients Who Received Placebo During 24 Weeks (Study 1) 1 Includes the following adverse reactions that occurred more frequently in the AGAMREE group than in placebo: abnormal behavior, aggression, agitation, anxiety, irritability, mood altered, sleep disorder, and stereotypy. Adverse Reaction AGAMREE 2 mg/kg/d (N=30) % AGAMREE 6 mg/kg/d (N=28) % Placebo (N=29) % Cushingoid Features 7 29 0 Psychiatric disorders 1 7 21 14 Vomiting 17 14 7 Weight increased 0 11 3 Vitamin D deficiency 7 11 0 Cough 10 7 3 Headache 7 7 3 Diarrhea 3 7 3 Increased appetite 3 7 3 Rhinitis 3 7 3 In a separate open-label safety study of pediatric patients aged 2 to less than 4 years (n=16) and pediatric patients aged 7 to less than 18 years (n=16) with DMD, adverse reactions were similar to those seen in the Study 1 pediatric patients.
🔄 Drug Interactions ▾
7 DRUG INTERACTIONS Strong CYP3A4 inhibitors: The maximum recommended daily dose is 4 mg/kg up to a maximum daily dosage of 200 mg for patients weighing more than 50 kg. ( 2.6 , 7.1 )
7.1Effect of Other Drugs on Vamorolone Co-administration of AGAMREE with itraconazole, a strong CYP3A4 inhibitor, increases vamorolone exposure [see Clinical Pharmacology ( 12.3 )] . Reduce the dosage of AGAMREE in patients when strong CYP3A4 inhibitors are used concomitantly [see Dosage and Administration ( 2.6 )] . No dosage adjustments are required when AGAMREE is concomitantly administered with moderate or weak CYP3A4 inhibitors.
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS
8.1Pregnancy 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.
An increase in embryofetal death, intrauterine growth retardation, and constriction of the ductus arteriosus were observed in some animal species. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Clinical Considerations Fetal/Neonatal Adverse Reactions Hypoadrenalism may occur in infants born to mothers receiving corticosteroids during pregnancy.
Infants should be carefully observed for signs of hypoadrenalism, such as poor feeding, irritability, weakness, and vomiting, and managed accordingly [see Warnings and Precautions ( 5.1 )]. Data Human Data Multiple cohort and case-controlled studies in humans suggest that maternal corticosteroid use during the first trimester increases the rate of cleft lip, with or without cleft palate, from about 1/1000 infants to 3-5/1000 infants. Two prospective case-controlled studies showed decreased birth weight in infants exposed to maternal corticosteroids in utero.
8.2Lactation Risk Summary There are no data on the presence of vamorolone in human milk or the effects on milk production. AGAMREE is indicated for use for the treatment of DMD, which is a disease of young male patients. However, systemically administered corticosteroids appear in human milk and could suppress growth, interfere with endogenous corticosteroid production, or cause other untoward effects.
The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need and any potential adverse effects on the breastfed infant.
8.4Pediatric Use The safety and effectiveness of AGAMREE for the treatment of DMD have been established in patients 2 years of age and older. Use of AGAMREE in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 121 males 4 to less than 7 years of age [see Clinical Studies ( 14 )]. Use of AGAMREE in patients 2 years to less than 4 years of age and 7 to less than 18 years of age is supported by findings of efficacy and safety in patients 4 to less than 7 years of age with DMD, and by pharmacokinetic and safety data from patients 2 to 4 years of age and 7 to less than 18 years of age [see Adverse Reactions ( 6.1 ) and Clinical Pharmacology ( 12.3 )] .
The safety and effectiveness in pediatric patients below the age of 2 years have not been established. Juvenile Animal Toxicity Data Oral administration of vamorolone (0, 15, 30, or 100 mg/kg/day) to juvenile mice from postnatal days 21 t…
🤰 Pregnancy ▾
8.1Pregnancy 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.
An increase in embryofetal death, intrauterine growth retardation, and constriction of the ductus arteriosus were observed in some animal species. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Clinical Considerations Fetal/Neonatal Adverse Reactions Hypoadrenalism may occur in infants born to mothers receiving corticosteroids during pregnancy.
Infants should be carefully observed for signs of hypoadrenalism, such as poor feeding, irritability, weakness, and vomiting, and managed accordingly [see Warnings and Precautions ( 5.1 )]. Data Human Data Multiple cohort and case-controlled studies in humans suggest that maternal corticosteroid use during the first trimester increases the rate of cleft lip, with or without cleft palate, from about 1/1000 infants to 3-5/1000 infants. Two prospective case-controlled studies showed decreased birth weight in infants exposed to maternal corticosteroids in utero.
🧒 Pediatric Use ▾
8.4Pediatric Use The safety and effectiveness of AGAMREE for the treatment of DMD have been established in patients 2 years of age and older. Use of AGAMREE in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 121 males 4 to less than 7 years of age [see Clinical Studies ( 14 )]. Use of AGAMREE in patients 2 years to less than 4 years of age and 7 to less than 18 years of age is supported by findings of efficacy and safety in patients 4 to less than 7 years of age with DMD, and by pharmacokinetic and safety data from patients 2 to 4 years of age and 7 to less than 18 years of age [see Adverse Reactions ( 6.1 ) and Clinical Pharmacology ( 12.3 )] .
The safety and effectiveness in pediatric patients below the age of 2 years have not been established. Juvenile Animal Toxicity Data Oral administration of vamorolone (0, 15, 30, or 100 mg/kg/day) to juvenile mice from postnatal days 21 to 81 resulted in no adverse effects on neurobehavioral function, sexual maturation, or reproductive function. Atrophy of the adrenal cortex, degeneration/necrosis of the liver, and decreased lymphocytes in lymphatic tissues were observed at all doses.
A no-effect dose for general toxicity was not identified. Plasma exposures (AUC) at the lowest dose tested (15 mg/kg/day) were lower than that in humans at the maximum recommended human dose (300 mg/day).
🧓 Geriatric Use ▾
8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with AGAMREE.
🆘 Overdosage ▾
10 OVERDOSAGE Treatment of acute overdosage of vamorolone is by immediate supportive and symptomatic therapy. Gastric lavage or emesis can be considered.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action 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.
12.2Pharmacodynamics Vamorolone produced a dose-dependent decrease in morning cortisol levels in the clinical studies. Treatment with corticosteroids is associated with a suppression of endogenous cortisol concentrations and an impairment of the hypothalamus-pituitary-adrenal (HPA) axis function. A dose-dependent increase in leukocyte counts and lymphocyte counts was observed in clinical studies with vamorolone.
Cardiac Electrophysiology Vamorolone does not cause a mean increase in the QTc interval >20 milliseconds (ms) at 1.6 times the approved recommended dose.
12.3Pharmacokinetics 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. Vamorolone does not accumulate with repeated administration after once daily dosing.
Absorption After oral administration with food, the median T max is about 2 hours (range 0.5 to 5 hours). Effect of Food Co-administration of vamorolone (2 mg/kg) with a high-fat/high-calorie meal reduced C max by 18%, increased AUC by 13%, and delayed T max by one hour. Co-administration of vamorolone (2 mg/kg) with a low-fat/low-calorie meal reduced C max by 4%, increased AUC by 14%, and delayed T max by one hour [see Dosage and Administration ( 2.2 )].
Distribution The apparent volume of distribution of vamorolone for a DMD patient with a body weight of 20 kg taking AGAMREE with a meal is 162 L based on the population PK analysis. Protein binding is 88.1% in vitro. The blood to plasma ratio is approximately 0.87.
Elimination Vamorolone clearance for a DMD patient with a body weight of 20 kg taking AGAMREE with a meal is 58 L/h based on the population PK analysis. The terminal elimination half-life of vamorolone is approximately 2 hours. Metabolism Vamorolone is metabolized via multiple Phase I and Phase II metabolic pathways, such as glucuronidation, hydroxylation, and reduction.
The main plasma and urine metabolites are formed through direct glucuronidation as well as hydrogenation with subsequent glucuronidation. Metabolism of vamorolone is mediated through CYP3A4/5, CYP2C8, UGT1A3, UGT2B7, and UGT2B17. Excretion Approximately 30% of vamorolone dose is excreted in feces (15.4% unchanged) and 48% of vamorolone dose is excreted in urine as metabolites (<1% unchanged).
The major metabolites in urine are glucuronides. Specific Populations No clinically significant differences in the pharmacokinetics of vamorolone were observed based on race and sex. Pediatric Patients In children ages 4 to 7 years (N=12) who were administered 6 mg/kg of AGAMREE daily, on Day 1 and Day 14, the C max values (arithmetic mean, SD) of vamorolone were 856 ng/mL (471) and 970 ng/mL (270), respectively, and the AUC 24 values (arithmetic mean, SD) of vamorolone were 3279 ng·h/mL (1693) and 3606 ng·h/mL (897), respectively.
The pharmacokinetics of vamorolone was also characterized in DMD children ages 2 to 4 (N=6). Similar PK parameters were observed in younger children after administration of 6 mg/kg AGAMREE when compared to older children. Patients with Hepatic Impairment In a clinical study (N=16), vamorolone C max and AUC 0inf values were increased by approximately 1.7- and 2.6-fold, respectively, in subjects with moderate hepatic impairment (Child-Pugh Class B) compared with healthy matched controls [see Dosage and Administration ( 2.3 ) and Use in Specific Populations ( 8.6 )] .
There is no experience with vamorolone in patients with severe hepatic impairment. Drug Interaction Studies Effect of Strong CYP3A4 Inhibitors on Vamorolone Compared to adminis…
🧬 Mechanism of Action ▾
12.1Mechanism of Action 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.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1How Supplied AGAMREE oral suspension is an orange flavored homogeneous white to off-white suspension, containing 40 mg/mL of vamorolone. AGAMREE is supplied as 100 mL in 125 mL glass bottle packaged with one bottle adapter, two 5 mL oral syringes, and an Instructions for Use: NDC 69616-264-38.
16.2Storage and Handling Store the bottle upright at room temperature between 20°C to 25°C (68°F to 77°F). Excursions permitted between 15°C to 30°C (59°F to 86°F) in the original carton. See USP controlled room temperature. After opening, store the bottle upright in a refrigerator 2°C to 8°C (36°F to 46°F). Do not freeze. Discard any unused AGAMREE oral suspension remaining after 3 months of first opening the bottle.
16.1How Supplied AGAMREE oral suspension is an orange flavored homogeneous white to off-white suspension, containing 40 mg/mL of vamorolone. AGAMREE is supplied as 100 mL in 125 mL glass bottle packaged with one bottle adapter, two 5 mL oral syringes, and an Instructions for Use: NDC 69616-264-38.
📦 Storage and Handling ▾
16.2Storage and Handling Store the bottle upright at room temperature between 20°C to 25°C (68°F to 77°F). Excursions permitted between 15°C to 30°C (59°F to 86°F) in the original carton. See USP controlled room temperature. After opening, store the bottle upright in a refrigerator 2°C to 8°C (36°F to 46°F). Do not freeze. Discard any unused AGAMREE oral suspension remaining after 3 months of first opening the bottle.
📋 Description ▾
11 DESCRIPTION AGAMREE (vamorolone) oral suspension contains vamorolone, a corticosteroid. Vamorolone [17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione] is a white to off-white powder with a molecular formula of C 22 H 28 O 4 and a molecular weight of 356.46 g/mol. Its structural formula is: Vamorolone is freely soluble in methanol and dioxane and sparingly soluble in ethanol and acetone.
AGAMREE for oral administration is available as an oral suspension in a strength of 40 mg/mL. The oral suspension contains vamorolone and the following inactive ingredients: citric acid (monohydrate), disodium phosphate, glycerin, hydrochloric acid (for pH adjustment), orange flavor, sodium benzoate, sucralose, water, and xanthan gum. Structural Formula
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION Advise the patients and/or caregivers to read the FDA-approved patient labeling ( Instructions for Use ). Administration Warn patients and/or caregivers to not stop taking AGAMREE abruptly or without first checking with their healthcare providers as there may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis [see Dosage and Administration ( 2.7 ) and Warnings and Precautions ( 5.1 )]. AGAMREE oral suspension should be taken once daily, preferably with a meal.
AGAMREE oral suspension must be shaken well for about 30 seconds prior to measuring out each dose with the enclosed oral syringe. Discard any unused AGAMREE oral suspension remaining after 3 months of first opening the bottle. Immunosuppression and Increased Risk of Infection Tell patients and/or caregivers to inform their healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine.
Medical advice should be sought immediately if the patient develops fever or other signs of infection. Patients and/or caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed [see Warnings and Precautions ( 5.2 )] .
Alterations in Cardiovascular/Renal Function Inform patients and/or caregivers that corticosteroids, including AGAMREE, can cause an increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed [see Warnings and Precautions ( 5.3 )]. Behavioral and Mood Disturbances Advise patients and/or caregivers about the potential for severe behavioral and mood changes with AGAMREE and encourage them to seek medical attention if psychiatric symptoms develop [see Warnings and Precautions ( 5.5 )].
Decreases in Bone Mineral Density Advise patients and/or caregivers about the risk of osteoporosis with prolonged use of AGAMREE, which can predispose the patient to vertebral and long bone fractures [see Warnings and Precautions ( 5.6 )]. Ophthalmic Effects Inform patients and/or caregivers that AGAMREE may cause cataracts or glaucoma and advise monitoring if administered for more than 6 weeks [see Warnings and Precautions ( 5.7 )]. Vaccination Advise patients and/or caregivers to bring immunizations up-to-date according to immunization guidelines prior to starting therapy with AGAMREE.
Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting AGAMREE. Inform patients and/or caregivers that they may receive concurrent vaccinations with use of AGAMREE, except for live-attenuated or live vaccines [see Warnings and Precautions ( 5.8 )]. Drug Interactions Certain medications can cause an interaction with AGAMREE [see Drug Interactions ( 7.1 )] .
Advise patients and/or caregivers to inform their healthcare provider of all the medicines the patient is taking, including over-the-counter medicines (such as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Inform patients and/or caregivers that alternate therapy, dosage adjustment, and/or special test(s) may be needed during the treatment. Manufactured for: Catalyst Pharmaceuticals, Inc.
Coral Gables, FL 33134, USA AGAMREE Oral Suspension made in Italy. AGAMREE ® is a registered trademark of Santhera Pharmaceuticals (Schweiz) AG. 6010 Rev.
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