Cash PositionThe sale of the Rare Pediatric Disease Priority Review Voucher for $150M provides a significant infusion of non-dilutive capital to strengthen the company's balance sheet.
Financial PerformanceSales of Miplyffa for Niemann-Pick Type C came in higher than expected, indicating strong market performance.
Regulatory ApprovalFDA approval of Miplyffa marks it as the first approved therapy for Niemann-Pick disease type C in the US, potentially allowing for rapid adoption among patients.