Drug updated on 10/3/2024
| Dosage Form | Capsule (oral; 47 mg, 62 mg, 93 mg and 124 mg) |
| Drug Class | Heat shock protein stimulators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
Latest News
Summary
- This summary is based on the review of one randomized controlled trial(s). [1]
- At 12 months, arimoclomol showed a mean NPCCSS score progression of 0.76 compared to 2.15 for placebo, with a treatment difference of -1.40 (95% CI: -2.76, -0.03; P = .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.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Miplyffa (arimoclomol) Prescribing Information. | 2024 | Zevra Therapeutics, Inc. Celebration, FL |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. | 50Subjects F: 52% M: 48% | 2021 | Journal of Inherited Metabolic Disease |
Sex Distribution:
F:52%
M:48%
50Subjects
Year:
2021
Source:Journal of Inherited Metabolic Disease