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Cipaglucosidase alfa-atga

(Pombiliti®)

Pombiliti®

Drug updated on 10/21/2024

Dosage FormInjection (intravenous; 105 mg in a single-dose vial)
Drug ClassHydrolytic 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).

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Summary
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  • 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 (95% CI, 0.10-0.31; I² = 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.
  • Document 1 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 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.