Zevra Therapeutics to Present at the 21st Annual WORLDSymposium™

Core Points - Zevra Therapeutics will receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium, recognizing advancements in treating lysosomal diseases [2][3] - Eight abstracts related to MIPLYFFA have been accepted for presentation, including one selected for oral presentation by Dr. Eugen Mengel [1][4] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on rare disease therapeutics, aiming to develop transformational therapies for diseases with limited treatment options [21] - The company emphasizes a data-driven approach to overcome drug development challenges and meet patient needs [21] Product Information - MIPLYFFA (arimoclomol) is indicated for treating neurological manifestations of Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat [10] - The drug has received multiple designations from the FDA, including Breakthrough Therapy and Orphan Drug designations, highlighting its significance in treating NPC [9] Clinical Research and Presentations - The pivotal phase 3 trial of MIPLYFFA demonstrated that it halted disease progression compared to placebo over a one-year period, as measured by the NPC Clinical Severity Scale [9] - The WORLDSymposium will feature various presentations on MIPLYFFA, including efficacy results from a 12-month double-blind randomized trial [4][5][6] Event Participation - Zevra will participate in the WORLDSymposium taking place from February 3-7, 2025, in San Diego, CA, where attendees can visit their booth [7][8]