INTRODUCTION: Duchenne muscular dystrophy (DMD) results in progressive muscle loss as a result of dystrophin gene mutations. Most patients become non-ambulatory in their second decade, gradually losing pulmonary function and requiring non-invasive ventilation, with increasing risk of cardiac failure. Viltolarsen, an FDA-approved therapy for patients with DMD mutations amenable to exon 53 skipping, has been evaluated in both ambulatory and non-ambulatory patients during the Phase II clinical trial, GALACTIC53. However, real-world data for older, non-ambulatory patients remain limited. This analysis utilizes US claims data to assess the demographic, treatment, and care patterns in non-ambulatory patients with DMD aged ≥12 years when initiating viltolarsen.
METHODS: This retrospective cohort study will identify males aged ≥12 years with confirmed DMD diagnosis, a wheelchair or wheelchair-related claim(s), and anonymized open viltolarsen claims between January 1, 2017, and October 14, 2024 (to allow for minimum 12-month follow-up period for persistence analysis), from Veeva Compass Patient (longitudinal anonymous patient-level data). Analyses will include age at initiation, corticosteroid usage, proportion receiving viltolarsen as first exon-skipping therapy, concomitant DMD therapy usage, and treatment persistence. Cardiac medication usage and pulmonary equipment claims prior to treatment with viltolarsen will also be analyzed.
RESULTS: Key findings will characterize real-world viltolarsen treatment patterns in the older non-ambulatory population.
CONCLUSION: These findings will address an important evidence gap in the clinical management of late, non-ambulatory patients with DMD who are initiating viltolarsen. The results will help clinicians optimize care strategies and guide future research toward personalized treatment approaches for this complex patient population.