Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• Arimoclomol in Niemann–Pick Disease Type C: At 12 months, arimoclomol demonstrated a mean Niemann-Pick Disease Type C Clinical Severity Scale (NPCCSS) score progression of 0.76 compared to 2.15 for placebo, with a treatment difference of -1.40 (95% confidence interval (CI), -2.76 to -0.03; P = 0.046), reflecting a 65% reduction in disease progression.
• In patients receiving miglustat, arimoclomol resulted in stabilization of disease severity, with a treatment difference of -2.06 (P = .006), indicating significant benefit compared to placebo.
• In the arimoclomol group, 88.2% of patients (30 out of 34) experienced adverse events, with serious adverse events occurring in 14.7% of patients (5 out of 34). Treatment-related serious adverse events included urticaria and angioedema (n = 2).
• In the placebo group, 75.0% of patients (12 out of 16) experienced adverse events, and serious adverse events occurred in 31.3% of patients (5 out of 16).
• The study population included patients aged 2-18 years with Niemann-Pick disease type C (NPC), and a subgroup analysis showed that arimoclomol significantly stabilized disease severity over 12 months for patients receiving miglustat as part of their routine care.