Atidarsagene autotemcel (OTL-200 or “arsa-cel,” brand name Libmeldy in Europe) is a gene therapy for MLD targeting the ARSA gene. This single-use gene therapy is currently under FDA review, with a decision expected by March 18, 2024. The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the clinical and economic outcomes of arsa-cel for early-onset (LI and EJ) forms of MLD. This report presents a summary of our findings and highlights the key policy recommendations discussed at a public meeting of the California Technology Assessment Forum (CTAF) on September 29, 2023. The full report is available at https://icer.org/wp-content/uploads/2023/10/MLD-Final-Evidence-Report_For-Publication_10302023.pdf.
