Lumizyme is approved for the treatment of infantile-onset Pompe disease (IOPD)1
By providing a replacement for the missing or deficient GAA enzyme, Lumizyme can be an important part of a care plan for patients with IOPD.
The efficacy of Lumizyme has been confirmed in 3 clinical trials1
Three separate clinical trials assessed the safety and efficacy of Lumizyme, totaling 57 treatment-naive IOPD patients, aged 0.2 months to 3.5 years at first infusion.
IOPD studies overview
| Study 1 | Study 2 | Study 3 | |
| Study Design |
International, multicenter, open-label trial (N=18) Patient age at baseline: ≤7 months Dosing: 20 mg/kg or 40 mg/kg EOW Treatment duration: 52-106 weeks Outcome measure: Proportion of patients who died or needed invasive ventilator support at 18 months of age vs the mortality experience of a similar historical cohort of untreated patients |
International, multicenter, non-randomized, open-label clinical trial (N=21) Patient age at baseline: 3 months–3.5 years CRIM positive: n=18 Dosing: 20 mg/kg EOW Primary outcome measure: Proportion of patients alive at conclusion of treatment |
Open-label, single-center trial of patients identified through newborn screening (N=18) Patient age at first infusion: <6 months (0.2–5.8 months) CRIM positive: N=18 |
| Results |
At 18 months, all patients treated with Lumizyme were alive vs 1/61 (2%) of the historical control cohort. 15/18 (83%) Lumizyme patients were alive without ventilatory support. 3/18 (17%) required invasive ventilation. | At the 52-week interim analysis, 16/21 patients were alive. | At the time of the analysis, 16/18 patients reached 18 months of age without ventilator support. |
EOW=every other week.
Overall safety profile from IOPD trials
Adverse reactions that occurred in >5% of IOPD patients treated with Lumizyme in clinical trials.1
- The most serious adverse reactions reported with Lumizyme included anaphylaxis and acute cardiorespiratory failure
- The most common adverse reactions requiring intervention in clinical trials were hypersensitivity reactions, occurring in 20/39 (51%) patients treated with Lumizyme, and included rash, pyrexia, urticaria, flushing, decreased oxygen saturation, cough, tachypnea, tachycardia, hypertension/increased blood pressure, pallor, rigors, vomiting, cyanosis, agitation, and tremor. These reactions were more likely to occur with higher infusion rates
- Some patients who were pretreated with antihistamines, antipyretics, and/or corticosteroids still experienced hypersensitivity reactions
Adverse Reaction |
Number of patients |
| Hypersensitivity reactions | 20 (51) |
| Rash (including rash erythematous, rash macular, and maculopapular) | 7 (18) |
| Pyrexia | 6 (15) |
| Urticaria | 5 (13) |
| Flushing | 5 (13) |
| Hypertension/increased blood pressure | 4 (10) |
| Decreased oxygen saturation | 3 (8) |
| Cough | 3 (8) |
| Tachypea | 3 (8) |
| Tachycardia | 3 (8) |
| Erythema | 2 (5) |
| Vomiting | 2 (5) |
| Rigors | 2 (5) |
| Pallor | 2 (5) |
| Cyanosis | 2 (5) |
| Agitation | 2 (5) |
| Tremor | 2 (5) |
LOPD=late-onset Pompe disease.
Indication
Reference: 1. Lumizyme (alglucosidase alfa). Prescribing information. Genzyme Corporation, Cambridge, MA.
