Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 105 mg in a single-dose vial) |
| Drug Class | Hydrolytic lysosomal glycogen-specific enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing 40 kg and who are not improving on their current enzyme replacement therapy (ERT).
Latest News
Summary
- This summary is based on the review of two randomized controlled trial(s). [1-2]
- Nirmatrelvir/ritonavir significantly decreased COVID-19 hospitalization compared to placebo, with a relative risk (RR) of 0.17, with a 95% confidence interval (CI) of 0.10 to 0.31 and an I² of 77.2% from randomized controlled trials (RCTs) involving 3,542 persons, and an RR of 0.48 (95% CI, 0.37-0.60; I² = 95.0%) from 11 real-world studies (RWS) with 1,421,398 persons.
- In RCTs, nirmatrelvir/ritonavir showed no significant effect on all-cause mortality (RR = 0.27; 95% CI, 0.04-1.70; I² = 49.9%; 3 RCTs, 3,806 persons), while RWS indicated a significant reduction in mortality (RR = 0.24; 95% CI, 0.14-0.34; I² = 65%; 7 RWS, 286,131 persons).
- The composite outcome of hospitalization or mortality was significantly reduced by nirmatrelvir/ritonavir, with an RR of 0.62 (95% CI 0.55-0.70; I² = 0%), indicating moderate certainty in the evidence.
- There was no significant difference in viral clearance (RR = 1.19; 95% CI, 0.93-1.51; I² = 82%; 2 RCTs, 528 persons) or worsening severity (RR = 0.82; 95% CI, 0.66-1.01; I² = 47.5%; 3 RCTs, 1,824 persons).
- In the Phase 3 trial, 96% of patients receiving cipaglucosidase alfa-atga experienced at least one treatment-emergent adverse event, with common adverse events including fall, headache, nasopharyngitis, myalgia, and arthralgia; serious adverse events included 12 cases in the cipaglucosidase alfa-atga group, one of which was anaphylaxis deemed related to the drug.
- One study reported that three patients discontinued treatment due to infusion-associated reactions, with no new safety signals identified, indicating that while adverse events were common, most were not serious and did not lead to discontinuation in the majority of cases.
- The studies focused on adult patients (age 18 years or older) with late-onset Pompe disease, reporting separate outcomes for Enzyme Replacement Therapy (ERT)-experienced and ERT-naive patients; ERT-naive patients demonstrated greater improvements in 6-minute walk distance (6MWD) but experienced a more significant decline in forced vital capacity (FVC) compared to ERT-experienced patients, indicating varying responses to treatment based on previous ERT exposure.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Pombiliti (cipaglucosidase alfa-atga) Prescribing Information. | 2024 | Amicus Therapeutics US |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) | 118Subjects F: 56% M: 44% | 2024 | Journal of Neurology |
| Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial | 123Subjects F: 54% M: 46% | 2021 | The Lancet Neurology |
Sex Distribution:
F:56%
M:44%
118Subjects
Year:
2024
Source:Journal of Neurology
Sex Distribution:
F:54%
M:46%
123Subjects
Year:
2021
Source:The Lancet Neurology