Zevra Launches New Disease State Awareness Campaign, 'Learn NPC, Read Between the Signs,' to Drive Early Recognition and Treatment of Niemann-Pick Disease Type C

Core Points - Zevra Therapeutics launched a new awareness campaign titled 'Learn NPC, Read Between the Signs' on Rare Disease Day to promote early recognition and diagnosis of Niemann-Pick disease type C (NPC) [1][2][3] - The campaign aims to provide educational resources and testing information to healthcare professionals and treatment teams in the U.S. [2][3] - MIPLYFFA (arimoclomol), Zevra's approved therapy for NPC, was highlighted, which received FDA approval on September 20, 2024, and has shown to halt disease progression in clinical trials [4][5] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases with significant unmet needs [15] - The company emphasizes a data-driven approach to drug development and commercialization to address challenges in bringing new therapies to the market [15] Disease Information - Niemann-Pick disease type C (NPC) is characterized as an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder, leading to cholesterol accumulation in cells, particularly neurons [14] - The disease can affect both children and adults, presenting with varying symptoms and often resulting in irreversible disease progression and early mortality [14]