Zevra Therapeutics to Present Multiple Datasets on MIPLYFFA at ICIEM Congress

Zevra Therapeutics will present four posters on MIPLYFFA (arimoclomol) at the International Congress of Inborn Errors of Metabolism. The posters will detail positive data from a pediatric substudy, a new prespecified efficacy analysis, and a real-world setting study. MIPLYFFA is approved for the treatment of Niemann-Pick disease type C and has received Orphan Medicinal Product designation by the European Medicines Agency.

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) will present four posters on MIPLYFFA® (arimoclomol) at the International Congress of Inborn Errors of Metabolism (ICIEM), taking place from September 2 to 6, 2025, in Kyoto, Japan. The presentations will highlight the drug's efficacy and safety in various clinical settings, including a pediatric substudy and a real-world setting study.

MIPLYFFA is approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC), a rare genetic disorder that affects the metabolism of cholesterol and other lipids. The drug has received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.

One of the posters, titled "Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to 24 months at study enrollment," will present new data from a multi-center pediatric substudy in patients younger than two years old. This study aims to evaluate the safety and efficacy of MIPLYFFA in the youngest population affected by NPC.

Another poster, "Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network," will detail the drug's mechanism of action. This poster was selected for a Best Poster award, highlighting the significance of the findings.

A third poster, "Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United States," will present data from a real-world setting study. This study will provide insights into the long-term outcomes of patients treated with MIPLYFFA under routine clinical care.

The fourth poster, "Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for treatment of Niemann-Pick disease type C in patients treated with miglustat," will present data from a 12-month double-blind randomized trial and an open-label extension phase. This study will evaluate the efficacy of MIPLYFFA in combination with miglustat, a commonly used treatment for NPC.

These presentations will provide valuable insights into the clinical development of MIPLYFFA and its potential to improve the lives of patients with NPC. Zevra Therapeutics' extensive clinical development program for MIPLYFFA, which includes a Phase 2/3 clinical trial, an Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, is the most expansive in NPC to date.

[1] https://www.globenewswire.com/news-release/2025/08/28/3140697/16626/en/Zevra-Therapeutics-Announces-Multiple-Datasets-on-MIPLYFFA-arimoclomol-to-be-Presented-at-the-International-Congress-of-Inborn-Errors-of-Metabolism-ICIEM.html
[2] https://www.stocktitan.net/news/ZVRA/zevra-therapeutics-announces-multiple-datasets-on-miplyffa-1dqvnlsuw49b.html