- MIPLYFFA shows sustained efficacy in reducing Niemann-Pick disease type C (NPC) progression for at least 5 years.
- Over 270 patients worldwide have been treated with MIPLYFFA, some for up to 7 years, with no new safety concerns.
- The study's results align with previous trials, reinforcing MIPLYFFA's efficacy and safety profile.
Zevra Therapeutics (ZVRA, Financial) has released long-term efficacy data for its treatment, MIPLYFFA® (arimoclomol), in patients with Niemann-Pick disease type C (NPC), demonstrating its consistent ability to reduce disease progression. The findings, published in the journal Molecular Genetics and Metabolism, reveal that the open-label extension study, spanning 48 months, showed sustained benefits for at least 5 years, with some patients experiencing positive effects for up to 7 years.
The investigation included safety and efficacy data from over 270 patients globally, bolstering the evidence base for MIPLYFFA in treating this rare disease. The study utilized the 5- and rescored 4-domain NPC Clinical Severity Scale, the only validated tool for measuring NPC progression, confirming sustained benefits without any new safety issues.
MIPLYFFA, which was approved by the U.S. Food and Drug Administration in September 2024, operates by addressing lysosomal dysfunctions that lead to this neurodegenerative disease, offering a significant advancement over previous treatment options that were scarce.
This publication not only enhances MIPLYFFA's clinical profile but also potentially expands its application within the NPC patient community, representing a pivotal step forward in the management of this devastating condition. Zevra Therapeutics continues to focus on providing treatments for individuals with rare diseases, driven by their commitment to improving patient care.
