FDA approves first Niemann-Pick disease, type C treatment

Today, the US Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC).

Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children two years of age and older.

The FDA granted approval to US rare diseases company Zevra Therapeutics (Nasdaq: ZVRA) which developed Miplyffa, that is the first drug approved by the FDA to treat NPC.