Last reviewed 2026-04-20 · How we verify

Pombiliti (CIPAGLUCOSIDASE ALFA)

Pombiliti works by replacing the deficient acid alpha-glucosidase enzyme in patients with Late-onset Pompe disease.

At a glance

Mechanism of action

Imagine your cells have a broken recycling system, and Pombiliti is a new recycling truck that delivers the missing enzyme to help break down and recycle cellular waste. This helps restore normal cellular function and reduces the accumulation of toxic substances in the body.

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

Key clinical trials

• A Global Prospective Observational Registry of Patients With Pompe Disease
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) (PHASE3)
• A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (PHASE3)
• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (PHASE3)
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (PHASE1,PHASE2)
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PHASE3)

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 Therapeutics Europe Limited portfolio CI