FDA Approves First Treatment for Niemann-Pick Disease Type C

Zevra Therapeutics' Miplyffa, in combination with miglustat, is now approved to treat neurological symptoms in NPC patients aged 2 and older.

Miplyffa (arimoclomol) is the first FDA-approved drug specifically for Niemann-Pick disease type C (NPC).
NPC is a rare genetic disorder causing progressive neurological symptoms and organ dysfunction due to lipid transport issues within cells.
The approval follows a 12-month randomized, double-blind, placebo-controlled trial showing Miplyffa slowed disease progression.
The drug is approved for use alongside Johnson & Johnson's enzyme inhibitor Zavesca (miglustat) and can be administered three times daily.
Zevra Therapeutics plans to make Miplyffa commercially available in the U.S. within the next 8 to 12 weeks.