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. ...