Zevra’s Arimoclomol US FDA Panel Nod Includes Nonclinical Data Doubts

03 Aug 2024

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.

Confirm — Zevra's nonclinical data cannot provide confirmatory evidence. • Source: Shutterstock

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.

Key Takeaways

The advisory committee majority voted that the pivotal trial results and other clinical data supported arimoclomol’s efficacy in NPC.

Clinical data beyond the randomized, placebo-controlled study also provide reassuring confirmatory evidence of arimoclomol’s efficacy and safety, a majority of...