Background: Standard of care corticosteroids are known to slow progression and preserve both skeletal and cardiac muscle function in DMD. Vamorolone, a novel steroid, differs from classic corticosteroids in its chemical structure, retaining the potent anti-inflammatory effect but, in contrast, being an antagonist of the mineralocorticoid receptor.
Objectives/Methods: We conducted a retrospective, observational study using de-identified data from a proprietary specialty pharmacy database. Patients were included if they were male, ≤30 years of age, and had a DMD diagnosis and ≥1 adjudicated vamorolone dispense between December 2023 and May 2025. Descriptive statistics were used to evaluate patient demographics, patient/caregiver-reported prior and concomitant medications, and vamorolone utilization.
Results: Among 997 male patients initiating vamorolone, the mean age at enrollment was 13.0 years, with primary payers being Medicaid (50.9%) and commercial insurance (45.4%). Approximately 80% were receiving corticosteroids at vamorolone initiation, divided evenly between prednisone and deflazacort. Past gene therapy was reported in 12.0% and the use of exon-skipping agents and givinostat was reported in 18.0% and 11.1%, respectively. The mean starting dose of vamorolone was 4.8 mg/kg/d, used concomitantly with cardiac medication(s). Angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) use (renin-angiotensin system [RAS] inhibitors) was reported in 552 (55.4%) patients overall, with 519 (70.7%) at the clinical care guideline–recommended age of ≥10 years. Mineralocorticoid receptor antagonists (MRA) and beta-blockers were reported in 113 (11.3%) and 187 (18.8%) individuals, respectively. Overall, 220 (22.1%) and 57 (5.7%) patients reported receiving 2 and 3 distinct cardiac classes of medications, respectively, with 10.9% receiving concomitant RAS inhibition and MRA therapy.
Conclusions: Among patients with DMD receiving vamorolone, approximately two-thirds reported concomitant RAS inhibition at the guideline–recommended ≥10 years of age. As with any patient or caregiver reported analysis, limitations include a potential time delay, context of disease, and recall bias.