Drug updated on 4/17/2024
| Dosage Form | Injection (intravenous; 105 mg in a single-dose vial) |
| Drug Class | Hydrolytic lysosomal 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).
Summary
- Cipaglucosidase alfa-atga (Pombiliti) is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease who weigh at least 40 kg and who are not improving on their current enzyme replacement therapy.
- A Randomized Controlled Trial was reviewed, focusing on evaluating the safety and efficacy of Pombiliti in combination with Opfolda for treating adult patients with late-onset Pompe disease.
- The incidences of treatment-emergent adverse events between cipaglucosidase alfa plus miglustat (investigational therapy) and alglucosidase alfa plus placebo (control group) were similar. Serious adverse events occurred in both groups but only one such event was directly related to the investigational therapy.
- In terms of effectiveness, there was no significant difference between cipaglucosidase alfa plus miglustat and the standard treatment regimen in improving functional exercise capacity measured by a 6-minute walk distance after 52 weeks.
- The study population comprised adults suffering from late-onset Pompe disease, including those who had been receiving enzyme replacement therapy for at least two years as well as enzyme replacement therapy-naive patients. Further research should investigate the benefits in respiratory function and long-term effects among those who have been on enzyme replacement therapy for more than two years.
- Compared to other drugs like standard Alglucosidase Alfa Treatment, this investigational therapy presents an alternative without demonstrating superiority based on the primary outcome assessed during the trial period; however, potential subgroup differences or outcomes could benefit more, warranting further investigation.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Pombiliti (cipaglucosidase alfa-atga ) Prescribing Information. | 2023 | Amicus Therapeutics US |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| 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: 55% M: 45% | 2023 | The Lancet Neurology |
Sex Distribution:
F:55%
M:45%
123Subjects
Year:
2023
Source:The Lancet Neurology