Santhera Pharmaceuticals received approval from the U.S. Food and Drug Administration (FDA) for Agamree^® (vamorolone) oral suspension on Oct. 26, 2023. Agamree is a corticosteroid that decreases inflammation and immune function for patients aged two years and older who have Duchenne muscular dystrophy (DMD). At a recommended dose of 6mg/kg once a day, it should be taken along with food. The maximum daily dose is 300mg and doses may be lowered gradually to 2mg/kg/day depending on the patient’s response to therapy. Starting in the first quarter of 2024, Catalyst Pharmaceuticals will distribute Agamree in the United States exclusively through AnovoRx. Its cost has not yet been announced. For full prescribing information, look here.

At a Glance

• Brand (Generic) Name: Agamree (vamorolone)
• Manufacturer: Santhera Pharmaceuticals
• Date Approved: Oct. 26, 2023
• Indication: to treat DMD for patients two years old and older
• Dosage Forms Available: oral suspension containing 40mg/mL
• Launch Date: First quarter 2024
• Estimated Annual Cost: not yet available
• DMD is a rare genetic condition that affects around 10,000 patients, almost exclusively boys and young men, in the United States.
• In DMD, a mutation in the gene for dystrophin, a muscle protein, causes progressive muscle wasting — typically beginning with muscles in the shoulders and thighs, then spreading gradually to other muscles, including the heart. Eventually, DMD patients need to use wheelchairs and ventilators. Currently, few patients live beyond 30 years of age.
• Corticosteroids are the current standard of care for DMD.
• Like other corticosteroids, Agamree decreases both immune and inflammatory activity to relieve symptoms of DMD and also to delay the progression of the disease. It does not block the same enzyme, however, potentially reducing its side effects.
• In three open-label clinical trials, patients taking Agamree for as long as four years had similar symptom relief as patients who took the currently recommended corticosteroid treatment regimen for DMD.
• In the studies, Agamree was generally well tolerated. Side effects included deficiency of vitamin D and vomiting. No adverse effects (AEs) were seen on growth or bone metabolism.
• Emflaza^™ (deflazacort – Marathon Pharmaceuticals) is another oral corticosteroid that is FDA-indicated to treat DMD symptoms for patients who are five years old and older.
• In June 2023, the FDA approved a gene therapy, Elevidys^® (delandistrogene moxeparvovec-rokl) to treat DMD.
• Several injectable drugs are available in the U.S. to treat DMD that has specific mutations.
• Designated as an Orphan Drug for treating DMD, Agamree was approved under the FDA’s Fast Track program. It also was granted an FDA Rare Pediatric Disease Priority Review Voucher that can be used to expedite a future drug application or sold to another company.