Help patients start POMBILITI + OPFOLDA

Simple steps from prescription to delivery

Once you decide to prescribe POMBILITI™ (cipaglucosidase alfa-atga) + OPFOLDA™ (miglustat), here’s what happens next:

Once you complete the Patient Referral Form (PRF), send it to AMICUS ASSIST®

Fax number: 1-833-264-2873

A Case Manager will reach out to you after you prescribe POMBILITI + OPFOLDA and will also contact your patient

AMICUS ASSIST will:

Confirm if a Prior Authorization is required
Provide an explanation of the patient’s eligibility and potential financial assistance options
Provide additional reimbursement support

Depending on the treatment setting, AMICUS ASSIST will do one of the following:

Triage the Patient Referral Form to the Specialty Pharmacy (SP). The SP will work directly with the prescriber's office to confirm the prescription and fill requests for ancillary supplies
Contact the prescriber to review the ordering process for Direct Purchase orders

AMICUS ASSIST will help manage the POMBILITI + OPFOLDA delivery based on site of care

Patient referral form

A form requiring patient and prescriber information to get patients started on POMBILITI + OPFOLDA

Download form

Additional Resources

POMBILITI + OPFOLDA Product Ordering Information

Provides NDC codes and information on pharmacy and distributor networks

Download

POMBILITI + OPFOLDA Billing & Coding Guide

A sample billing and coding guide with sample CMS‑1450 and CMS‑1500 claim forms

Download

Have your patients reach out to AMICUS ASSIST

AMICUS ASSIST is a program that provides dedicated support to individuals who have been prescribed an Amicus medication for an approved use. We strive to help patients navigate the financial and insurance-related aspects of their treatment while providing personalized assistance every step of the way.

Financial Support

We are committed to helping your patients get access to treatment.

AMICUS ASSIST is proud to help identify possible sources of financial assistance including:

Co-Pay Assistance*

Eligible commercially or privately insured patients may pay as little as $0 per month for treatment

Amicus Patient Assistance Program

For eligible uninsured or underinsured patients^†

Support from independent charitable foundations

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 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.

AMICUS ASSIST is a program that provides dedicated support to individuals who have been prescribed an Amicus medication. We strive to help patients navigate the financial and insurance-related aspects of their treatment while providing personalized assistance every step of the way. Call us toll-free at:

1-833-AMICUS-A (1-833-264-2872), Monday to Friday, 8AM – 8PM ET

Visit AmicusAssistHCP.com for additional resources

Important Safety Information

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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.

INDICATION

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.

Important Safety Information

Expand

Important Safety Information

WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

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).

WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

INFUSION-ASSOCIATED REACTIONS (IARs)

RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS

CONTRAINDICATION

POMBILITI in combination with OPFOLDA is contraindicated in pregnancy.

EMBRYO-FETAL TOXICITY

RISKS ASSOCIATED WITH POMBILITI AND OPFOLDA

POMBILITI and OPFOLDA must be administered in combination.

ADVERSE REACTIONS

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.

INDICATION

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