Last reviewed 2026-05-31 · How we verify

POMBILITI (CIPAGLUCOSIDASE ALFA-ATGA)

POMBILITI (generic name: CIPAGLUCOSIDASE ALFA-ATGA) is a drug developed by AMICUS THERAP US. It is currently FDA-approved for Late-onset Pompe disease.

Pompe Disease is a condition studied in clinical trials, with interventions including Cipaglucosidase alfa, Miglustat, Alglucosidase alfa, and Avalglucosidase alfa. Cipaglucosidase alfa is a glycogen hydrolytic enzyme used to treat Pompe Disease, classified as a hydrolytic enzyme with a molecular target of glycogen.

At a glance

Approved indications

• Late-onset Pompe disease

Boxed warnings

• WARNING: 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) ]. Risk of Acute Cardiorespiratory Failure in Susceptible Patients Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function for whom fluid restriction is indicated may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. More frequent monitoring of vitals should be performed during POMBILITI infusion in such patients [see Warnings and Precautions (5.3) ]. WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS See full prescribing information for complete boxed warning Hypersensitivity Reactions Including Anaphylaxis Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. ( 5.1 ) Infusion-Associated Reactions (IARs) If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. ( 5.2 ) Risk of Acute Cardiorespiratory Failure in Susceptible Patients Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. ( 5.3 )

Common side effects

• Headache
• Diarrhea
• Fatigue
• Nausea
• Abdominal pain
• Pyrexia
• Myalgia
• Arthralgia
• Increased blood pressure
• Pain
• Tremor
• Dyspepsia

Serious adverse events

• Anaphylaxis
• Urticaria
• Dyspnea
• Chest discomfort
• Hypotension
• Tachycardia
• Muscle spasms
• Dizziness
• Cough
• Vomiting

Key clinical trials

• A Global Prospective Observational Registry of Patients With Pompe Disease (N/A)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• POMBILITI CI brief — competitive landscape report
• POMBILITI updates RSS · CI watch RSS
• AMICUS THERAP US portfolio CI

Frequently asked questions about POMBILITI

Related

• Target: All drugs targeting Cation-independent mannose-6-phosphate receptor
• Manufacturer: AMICUS THERAP US — full pipeline
• Therapeutic area: All drugs in Other
• Indication: Drugs for Late-onset Pompe disease

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing