Real-world safety and effectiveness of arimoclomol in 109 individuals with Niemann-Pick disease Type C (NPC) enrolled in the US Early Access Program across 14 sites, followed for up to 4 years; 65.1% also received miglustat.
Disease severity (5DNPCCSS) remained stable over 4 years: mean scores were 11.2 at Baseline vs. 11.1 at Year 1 (n=78) and 11.0 at Baseline vs. 12.0 at Year 4 (n=22). Only 12.8% of participants (13/109) experienced treatment-related adverse events (17 events total out of 248).
No control arm for comparison; Year 4 cohort shrank to n=22, raising attrition/survivorship bias concerns; disease severity assessments were part of routine care rather than a standardized trial protocol.
Arimoclomol appears well-tolerated over nearly 2.5 years of mean exposure, with stable NPC severity scores in a real-world population including adults and those not on miglustat. Clinicians managing NPC patients can be reassured by this 4-year safety profile, especially when combining arimoclomol with miglustat.
