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# Zevra’s Niemann-Pick Disease Drug Approved
- URL: https://www.fdaweb.com/zevras-niemann-pick-disease-drug-approved/
- Published: 2024-09-20T12:00:00.000Z
- Updated: 2026-09-14T14:41:57.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157821

FDA has approved Zevra Therapeutics’ Miplyffa (arimoclomol) for treating Niemann-Pick disease, Type C (NPC) in adults and children aged two and older. The rare genetic disorder is characterized by progressive neurological deterioration and organ dysfunction, an [agency release](https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-niemann-pick-disease-type-c?utm%5Fmedium=email&utm%5Fsource=govdelivery) says.

The disease is caused by changes in either the NPC1 or NPC2 gene, affecting the transport of cholesterol and other lipids within a cell, FDA says. “As a result, these cells do not function as they should, ultimately causing organ damage,” it says. “On average, individuals affected by this devastating disease only live for about 13 years.”

Approval was based on data from a randomized, double-blind, placebo-controlled 12-month trial in patients two to 19 years of age who had a molecularly confirmed diagnosis of NPC. “Miplyffa’s efficacy was demonstrated by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) score in the patients who used miglustat as their background treatment,” the release says. “The R4DNPCCSS is a measure of NPC disease progression that looks at four items that patients with NPC, their caregivers and physicians have identified as most relevant including ambulation, speech, swallow and fine motor skills. Higher scores signify a greater severity of the disease. Compared to placebo, Miplyffa resulted in a slower disease progression as measured by the R4DNPCCSS score.”

On the safety side, the therapy’s prescribing information contains a warning for hypersensitivity reactions including hives and angioedema, adding that individuals experiencing these should stop using the drug, according to the release. “The most common side effects of Miplyffa include upper respiratory tract infection, diarrhea and decreased weight,” it says.