IntraBio Inc.

When Zevra Therapeutics, then known as KemPharm, bought arimoclomol for peanuts with its acquisition of Orphazyme in 2022, it looked like a great bit of business and further confirmation has come wi

The rare lysosomal storage disorder Niemann-Pick disease type C (NPC) now has two approved therapies as the US Food and Drug Administration gave a thumbs-up to IntraBio Inc. ’s modified amino acid Aq

Zevra Therapeutics Inc. received the first-ever US Food and Drug Administration approval in Niemann-Pick disease type C (NPC) for Miplyffa (arimoclomol), but faces some complicating factors: co-admin

The US Food and Drug Administration’s September will probably look quiet after August 2024 produced seven novel approvals and two complete response letters, but should still be interesting. As many as