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 Viewpoint - Zevra Therapeutics has launched a new awareness campaign, 'Learn NPC, Read Between the Signs,' on Rare Disease Day to promote early recognition and diagnosis of Niemann-Pick disease type C (NPC), an ultra-rare neurodegenerative disorder [2][3][4] Company Initiatives - The campaign aims to provide educational resources and testing information to healthcare professionals and treatment teams in the U.S. to improve the diagnosis of NPC [3][4] - Zevra's Chief Commercial Officer emphasized the company's commitment to addressing unmet needs in rare diseases and supporting the NPC community [3] Product Information - MIPLYFFA (arimoclomol) is Zevra's approved therapy for NPC, which received FDA approval on September 20, 2024 [5] - The drug works by activating transcription factors that regulate lysosomal expression and has shown to halt disease progression in a pivotal phase 3 trial [5] - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [6] Disease Overview - Niemann-Pick disease type C is characterized by the body's inability to transport cholesterol and lipids, leading to their accumulation in cells, particularly neurons [15] - The disease presents with varying symptoms and can lead to significant physical and cognitive limitations, often resulting in early mortality [15] Company Background - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to create transformational therapies through data-driven strategies [16]