Prescription medicine
Cipaglucosidase alfa Brand names: Pombiliti
Cipaglucosidase alfa (Pombiliti) is a prescription medicine. No generic is listed in FDA's NDC Directory.
- No generic listed
- Boxed warning
Check it at the source: the cipaglucosidase alfa FDA label on DailyMed (effective August 12, 2026); every cipaglucosidase alfa label on DailyMed.
Highlights of the record
FDA label of August 12, 2026; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026
SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS,.
and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS Hypersensitivity Reactions Including Anaphylaxis Patients treated with POMBILITI have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during POMBILITI administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, POMBILITI should be discontinued immediately, and appropriate medical treatment should be initiated. In patients with severe hypersensitivity reaction, desensitization measures to POMBILITI may be considered [see Warnings and Precautions (5.1) ]. Infusion-Associated Reactions (IARs) Patients treated with POMBILITI have experienced severe IARs. If severe IARs occur, immediately discontinue the POMBILITI infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering POMBILITI following severe IARs. Patients with an acute underlying illness at the time of POMBILITI infusion may be at greater risk for IARs. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from IARs [see Warnings and Precautions (5.2) ]. […]
Used for
From the label: indications and usagePOMBILITI is indicated, in combination with Opfolda, 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).
Forms and strengths
- 105 mg
Injection (powder) (lyophilized) for solution.
On the market
- Generic approvals
- No generic approved
- Labelers listed (NDC)
- 1 of which 0 under generic applications, 1 under brand applications, repackagers included
Supply and safety
- Pharmacy cost (NADAC)
- Not in the survey
- Shortage (FDA)
- No current entry
- Recalls by its makers (FDA)
- No recall recorded
- FAERS reports
- 85 through July 30, 2026
What is cipaglucosidase alfa used for?
From the label: indications and usagePOMBILITI is indicated, in combination with Opfolda, 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). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme 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).
FDA pharmacologic class: Hydrolytic Lysosomal Glycogen-specific Enzyme.
Source: FDA drug label (POMBILITI ATGA), AMICUS THERAPEUTICS US, LLC, effective August 12, 2026 (SPL set d77aa596-910d-516b-e053-2995a90a0839), via openFDA; the full label on DailyMed
Is there a generic for cipaglucosidase alfa (Pombiliti)?
No generic cipaglucosidase alfa is approved (Drugs@FDA, October 8, 2026).
Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)
What does the cipaglucosidase alfa label say about other medicines?
The cipaglucosidase alfa label names 1 other medicine in its interaction, contraindication or warning sections. The labels of 1 other medicine name cipaglucosidase alfa. The sentences are the labels' own; a label lists what its studies and reports found, not every interaction.
| Medicine named | Section | What the label says |
|---|---|---|
| Miglustat | contraindications | CONTRAINDICATIONS POMBILITI in combination with Opfolda is contraindicated in pregnancy [see Warnings and Precautions (5.4) and Use in Specific Populations (8.1) ]. |
Source: FDA drug label (POMBILITI ATGA), AMICUS THERAPEUTICS US, LLC, effective August 12, 2026 (SPL set d77aa596-910d-516b-e053-2995a90a0839), via openFDA; the full label on DailyMed
What do FAERS reports show for cipaglucosidase alfa?
The FDA Adverse Event Reporting System holds 85 reports that list cipaglucosidase alfa among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 9 are coded as a death and 16 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.
The most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026
What next?
- Other hydrolytic lysosomal glycogen-specific enzymes
- the pharmacologic class, with prices and generics
- Cipaglucosidase alfa side effects
- the label's list beside FAERS reports
- Check an interaction
- what two or more labels say about each other