Zevra Denmark A/S’ arimoclomol demonstrated a small but clinically meaningful treatment effect for Niemann-Pick disease type C (NPC) in the drug’s lone pivotal trial, a US Food and Drug Administration advisory committee said 2 August.
Zevra’s Arimoclomol US FDA Panel Nod Includes Nonclinical Data Doubts
The pivotal trial results, along with other data, support a conclusion of efficacy for Niemann-Pick disease type C, but nonclinical data are weak and cannot serve as confirmatory evidence for a single trial, the advisory committee majority said.
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