Zevra Therapeutics Announces Open Label Extension Data Showing Sustained Long-Term Efficacy of MIPLYFFA for the Treatment of NPC Published in the Journal of Molecular Genetics and Metabolism

Core Insights - Zevra Therapeutics announced the publication of long-term efficacy and safety results for MIPLYFFA (arimoclomol) in treating Niemann-Pick Disease Type C (NPC), demonstrating sustained disease progression halting over multiple years [1][2] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to provide transformational treatments for conditions with limited options [14] Product Information - MIPLYFFA is an approved therapy for NPC, indicated for use in combination with miglustat for patients aged 2 years and older [4] - The drug received FDA approval on September 20, 2024, and has shown the ability to activate transcription factors that regulate lysosomal function [3] Clinical Trial Results - The Phase 2/3 trial demonstrated that MIPLYFFA halted disease progression compared to placebo over a one-year duration, with long-term data showing sustained efficacy for at least five years in over 270 patients [2][3] - The open-label extension study confirmed no new safety concerns, aligning with previous trial results [2] Safety Information - Common adverse reactions reported include upper respiratory tract infection, diarrhea, and decreased weight, with hypersensitivity reactions noted in some patients [5][9][10] - MIPLYFFA may cause increases in serum creatinine without affecting glomerular function, primarily occurring in the first month of treatment [8]