Trial Profile

Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C

Status: Completed

Phase of Trial: Phase II/III

Latest Information Update: 31 Jul 2026

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At a glance

24 Jul 2026 According to a Zevra Therapeutics Media release, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease type C (NPC), a rare, progressive and life-threatening neurodegenerative disorder. Company plans to request re-examination following negative CHMP opinion for arimoclomol MAA.
23 Jun 2026 According to a Zevra Therapeutics Media release, data from the trial were presented in Molecular Genetics and Metabolism Reports.
23 Jun 2026 Results presented in the Zevra Therapeutics Media Release

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