- Publication validates MIPLYFFA’s mechanism of action in treating NPC disease
- Research demonstrates clear correlation between treatment and neurological improvements in animal models
- Data supports long-term clinical benefits observed in trials
Zevra Therapeutics (ZVRA, Financial) has announced the publication of important research in the journal Molecular Genetics and Metabolism. The study provides detailed insights into the mechanism of action of the drug MIPLYFFA®, which is used to treat Niemann-Pick disease type C (NPC). The research demonstrates how arimoclomol, the active ingredient in MIPLYFFA, enhances cellular processes crucial for improved lysosomal function.
The study highlights that arimoclomol enters cells and boosts the translocation of transcription factors EB and E3 (TFEB & TFE3) from the cytosol to the nucleus. This process results in the upregulation of CLEAR genes, including the crucial NPC1 gene, which plays a significant role in lysosomal function and cholesterol trafficking. Enhanced cholesterol trafficking was correlated with improved neurological behaviors such as rearing and gait in animal models.
Adrian Quartel, MD, FFPM, Chief Medical Officer at Zevra, emphasized that these insights mark a pivotal step in understanding MIPLYFFA’s interactions with NPC disease. The findings reinforce the drug’s efficacy in addressing the disease’s underlying pathology and support the long-term treatment benefits observed in clinical trials.
MIPLYFFA, which was approved by the U.S. Food and Drug Administration in September 2024, is used in combination with miglustat to manage the neurological manifestations of NPC in patients aged two years and older. The drug has also received Orphan Medicinal Product designation in the European Union, allowing for its broader use in treating NPC.