Objective: This retrospective real-world analysis aimed to describe the patient characteristics and early utilization patterns of VAM.
Methods: Data for this point in time analysis were sourced through a specialty pharmacy and stored in a data warehouse. The analysis included the first 100 patients to receive VAM from February 1 to March 30, 2024. Data were pooled to evaluate patient demographics, past and current DMD treatments, VAM dosing, geography and payer type.
Results: 100 patients met the criteria for on-label VAM utilization. Mean age of the analysis cohort at VAM initiation was 12.1 yrs with a mean weight of 45 kg. Seven boys received gene therapy prior to VAM initiation (mean age: 8 yr) and 16 patients were receiving exon skipping therapy at VAM initiation (mean age: 11.7 yrs). Prior CS use was common, with 43 and 42 patients receiving deflazacort and prednisone, respectively, and 16 naïve to CS therapy. The initial mean dose of VAM was 4.7 mg/kg/d (median: 5.45 mg/kg/d). The vast majority of patients (95%) were actively receiving VAM at the March 30, 2024 cutoff date, with 5 discontinuations (2 patient requests, 2 physician requests, and 1 adverse event). Payer types were 62% commercial, 35% Medicaid, and 3% other.
Conclusions: Among patients in the US receiving on-label doses of the novel steroid vamorolone, prior CS use was common, with a majority of patients switching from a traditional CS. Previous or current gene therapy or exon skipping therapy was not uncommon. The mean VAM starting dose correlated with a cohort of patients previously receiving CS therapy. Given the limitations of this retrospective analysis, these findings warrant additional review as VAM use increases.