FOR US HEALTHCARE
PROFESSIONALS ONLY
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).
If you’ve just prescribed POMBILITI + OPFOLDA, we can help streamline the next steps. Count on AMICUS ASSIST® to provide the education and support your patients may need to get the most out of their care.
Please click here for complete Important Safety Information including BOXED WARNING for severe hypersensitivity reactions, infusion-associated reactions, and risk of acute cardiorespiratory failure in susceptible patients.
Patient Education Liaisons (PELs) and Case Managers are available to assist your patients.
Monday–Friday, 8 AM–8 PM ET
Hablamos español. Administradores de casos de habla hispana están disponibles. Llamanos a +1-833-AMICUS-A (+1-833-264-2872)
Lunes–Viernes de 8 AM–8 PM ET
This is the first step to prescribe POMBILITI + OPFOLDA for your patient.
LOPD Referral Form Download
Discover how to get started and explore the process for completing your patient’s enrollment.
Process Navigation Tool Download
Once you’ve prescribed treatment and the patient has consented, an Amicus Patient Education Liaison (PEL) and Case Manager will be assigned to your patient.
Dedicated PELs can offer education and support to patients by:
Educating about LOPD and POMBILITI + OPFOLDA
Providing tips to help patients prepare for the treatment days
Helping patients have more productive conversations with their care team
PELs do not give medical advice or take the place of the patient's health care provider.
Case Managers can help patients navigate treatment access and financial assistance by:
Helping navigate their insurance coverage
Helping to coordinate prescription delivery
Identifying possible sources of financial assistance
With the combined knowledge and expertise of a PEL and a Case Manager, AMICUS ASSIST provides a two-pronged approach to ensure that your LOPD patients have access to the information and support they need.
We are committed to identifying possible sources of financial assistance for your patients, including:
Eligible, commercially or privately insured patients may pay as little as $0 per month for treatment
For eligible, uninsured, or underinsured patients†
AMICUS ASSIST can help identify independent charitable foundations who may be able to provide support. These foundations exist independently of Amicus and have their own eligibility criteria and application processes. Availability of support from the foundations is determined solely by the foundations.
*Co-pay assistance is only is valid for patients using commercial or private insurance for their Amicus medication. It is not valid for prescriptions reimbursed in whole or in part by Medicare, Medicaid, Veterans Administration, TRICARE, Department of Defense, similar federal or state programs, or where prohibited by law. Offer is only available for U.S. residents who have a valid prescription for an Amicus product. In accordance with state law, infusion-related costs are not covered for individuals residing in RI. Patients must be prescribed the medication for an approved use. Co-pay assistance is subject to annual maximum limits. Additionally, patients cannot seek reimbursement from their health insurance or other patient assistance programs for their co-pay. Amicus reserves the right to rescind, revoke, or amend this offer without notice.
†The Amicus Patient Assistance Program provides free product to those who qualify. Contact AMICUS ASSIST for details.
Amicus makes no representation or guarantee concerning reimbursement or coverage for any service or item. Information provided through the AMICUS ASSIST program does not constitute medical advice and is not intended to be a substitute for a consultation with a licensed healthcare provider or applicable third-party payer(s). Amicus reserves the right to modify the program at any time without notice.
All patients who have been prescribed an Amicus Therapeutics medication for an approved use are eligible for AMICUS ASSIST once a referral form has been submitted on their behalf. To get your patient started, simply complete and submit the Patient Referral Form, and then make sure your patient provides consent via the Patient Authorization form.
AMICUS ASSIST is pleased to help identify possible sources of financial assistance for your patients, including co-pay assistance, the Amicus Patient Assistance Program, and independent charitable foundations. For more information, please contact a Case Manager anytime Monday–Friday, 8 AM–8 PM ET.
Once you’ve submitted the Patient Referral Form, a Case Manager will contact your office about whether prior authorization (PA) is required, and the steps for obtaining authorization. For all other questions, please contact a Case Manager anytime Monday–Friday, 8 AM–8 PM ET.
To initiate the enrollment process, download the Patient Referral Form (PRF) today.
LOPD Referral Form Download
WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
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.
POMBILITI in combination with OPFOLDA 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).
Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information for OPFOLDA.
POMBILITI 
