Orphazyme A/S

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 US Food and Drug Administration maintained a high rate of complete response letter actions for novel agent applications in 2024, but recent history suggests that many of the 16 CRL recipients will

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