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ARTICLE | Product Development

Rare Disease Spotlight: Niemann-Pick pipeline behind two approvals

Therapies aim to enhance lysosomal function to clear cholesterol and glycosphingolipids, mostly via small molecules

October 24, 2024 10:56 PM UTC

It was a productive September for Niemann-Pick disease type C, bringing two FDA approvals and two clinical data updates for an ultra-rare genetic disease with no prior approved drugs. 

Miplyffa arimoclomol from Zevra Therapeutics Inc. (NASDAQ:ZVRA) was approved for NPC on Sept. 20, followed four days later by Aqneursa levacetylleucine from  IntraBio Inc. ...

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