HomeFelix Nunez2026-01-08T21:21:56+00:00

Ambassador Maddie, who was diagnosed with LOPD in 2009, singing into a microphone

Ambassador Maddie, who was diagnosed with LOPD in 2009, singing into a microphone

MORE THAN AN ERT

POMBILITI is a bis-m6P–enriched ERT.¹*
OPFOLDA is an enzyme stabilizer.²

This two-component therapy is designed to increase the amount of active enzyme available to the muscle.^2,3

See How POMBILITI + OPFOLDA Works

See 2 Years of Data

*Based on in vitro data. Cannot be translated to clinical outcomes.

REQUEST A REP

Our Rare Disease Specialists are ready to help.

START A PATIENT

Help your patients make the switch today

Opening double quotation mark icon

I wasn’t improving on the ERT I was taking so I decided it was time to make the switch to POMBILITI + OPFOLDA

— Sandi, a real patient on POMBILITI + OPFOLDA

Closing double quotation mark icon

7_years

of prior average ERT treatment before enrolling in the PROPEL study³*

90%

of patients enrolled in the
open-label extension (OLE) remained through Week 104^4†

* PROPEL: The first and only head-to-head, randomized, Phase 3 trial in LOPD with a majority ERT-experienced patient cohort.^1,2

^† 11 patients discontinued due to withdrawn consent, adverse events, investigator decision, or because they were lost to follow-up.⁴

^‡ Since launch as of July 2025.¹

Sign up for emails

Sign up to receive updates about LOPD and POMBILITI + OPFOLDA

ERT, enzyme replacement therapy; LOPD, late-onset Pompe disease.

IMPORTANT SAFETY INFORMATION

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. The risks and benefits of readministering POMBILITI following severe hypersensitivity reaction should be considered. If mild or moderate hypersensitivity reaction occurs, the infusion rate may be slowed or temporarily stopped. Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.

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. If mild or moderate IARs occur regardless of pretreatment, decreasing the infusion rate or temporarily stopping the infusion may ameliorate the symptoms. IARs may still occur in patients after receiving pretreatment.

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.

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.

CONTRAINDICATION
POMBILITI in combination with OPFOLDA is contraindicated in pregnancy.

EMBRYO-FETAL TOXICITY
Based on findings from animal reproduction studies, POMBILITI in combination with OPFOLDA may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. Verify the pregnancy status in females of reproductive potential prior to initiating treatment with POMBILITI in combination with OPFOLDA. Advise females of reproductive potential to use effective contraception during treatment with POMBILITI in combination with OPFOLDA and for at least 60 days after the last dose.

RISKS ASSOCIATED WITH POMBILITI AND OPFOLDA
POMBILITI and OPFOLDA must be administered in combination.

ADVERSE REACTIONS
The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with POMBILITI in combination with OPFOLDA in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.

To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

LACTATION
Advise females that breastfeeding is not recommended while on treatment with POMBILITI in combination with OPFOLDA.

References: 1. POMBILITI. Prescribing information. Amicus Therapeutics US, LLC; 2024. 2. OPFOLDA. Prescribing information. Amicus Therapeutics US, LLC; 2024. 3. Schoser B, Roberts M, Byrne BJ, et al. 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. Lancet Neurol. 2021;20(12):1027-1037. 4. Schoser B, Kishnani PS, Bratkovic D, et al. 104-week efficacy and safety of cipglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07). J Neurol. 2024;271(5):2810-2823 & supplemental material. 5. Amicus Therapeutics, Inc. Data on file.

All content of this website belongs to Amicus Therapeutics and is protected by copyright laws. All other intellectual property rights are reserved. The content may not be copied for commercial use or distribution or downloaded, modified, or posted to other sites.

This site is intended for US residents only and is governed solely by US laws and regulations. Amicus Therapeutics makes reasonable efforts to include accurate, up-to-date information on the site; however, it makes no warranties or representations as to its accuracy and does not assume liability for any errors or omissions.

POMBILITI^®, OPFOLDA^®, and AMICUS ASSIST^® are registered trademarks of Amicus Therapeutics, Inc.

© 2025 Amicus Therapeutics. All rights reserved. PP-AT-US-0001-0725-2 December 2025

IMPORTANT SAFETY INFORMATION

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. The risks and benefits of readministering POMBILITI following severe hypersensitivity reaction should be considered. If mild or moderate hypersensitivity reaction occurs, the infusion rate may be slowed or temporarily stopped. Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.

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. If mild or moderate IARs occur regardless of pretreatment, decreasing the infusion rate or temporarily stopping the infusion may ameliorate the symptoms. IARs may still occur in patients after receiving pretreatment.

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.

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.

CONTRAINDICATION

POMBILITI in combination with OPFOLDA is contraindicated in pregnancy.

EMBRYO-FETAL TOXICITY

Based on findings from animal reproduction studies, POMBILITI in combination with OPFOLDA may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. Verify the pregnancy status in females of reproductive potential prior to initiating treatment with POMBILITI in combination with OPFOLDA. Advise females of reproductive potential to use effective contraception during treatment with POMBILITI in combination with OPFOLDA and for at least 60 days after the last dose.

RISKS ASSOCIATED WITH POMBILITI AND OPFOLDA

POMBILITI and OPFOLDA must be administered in combination.

ADVERSE REACTIONS

The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with POMBILITI in combination with OPFOLDA in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.

To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

LACTATION

Advise females that breastfeeding is not recommended while on treatment with POMBILITI in combination with OPFOLDA.

Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information for OPFOLDA.

INDICATION

POMBILITI^® (cipaglucosidase alfa-atga) in combination with OPFOLDA^® (miglustat) is indicated 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).