A two-component therapy for adults with late-onset Pompe disease (LOPD) weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).

SUPPORT + RESOURCES helping you manage LOPD

EMMA
DIAGNOSED WITH LOPD IN 2009

SEE HOW IT ALL COMES TOGETHER

SEE HOW IT ALL COMES TOGETHER

To understand how POMBILITI + OPFOLDA work together, watch this video

To explore the clinical trial results for POMBILITI + OPFOLDA, watch this video

DOWNLOADABLE RESOURCES

PRODUCT BROCHURE

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TREATMENT DAY GUIDE

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INSURANCE 101 BROCHURE

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GET STARTED

Discuss

Talk to your doctor to see if POMBILITI + OPFOLDA could be right for you.

Download the Discussion Driver to help prepare for upcoming conversations with your care team.

PREPARE

Learn what to expect and get help managing your treatment day experience.

Download the MyDay Pompeโ„ข app from the App Store or Google Play.

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Connect

Find support.

AMICUS ASSISTยฎ provides comprehensive support to people prescribed POMBILITI + OPFOLDA. A Patient Education Liaison (PEL) and a Case Manager are standing by to help.

Get in touch with us by calling 1-833-AMICUS-A (1-833-264-2872).

Enjoy our AMICUS ASSIST Spotifyยฎ playlists.

Music. Meditations. Audiobooks. We’ve put together selections just for you. They can help you stay entertained, relax, or even learn something new.

Click the buttons to stream these free Spotify playlists.

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The following Pompe disease organizations and websites can help you connect with others while providing information that may be relevant to your experience.

Acid Maltase Deficiency Association (AMDA)

The AMDA assists in funding research and promoting public awareness of Pompe disease.

International Pompe Association

The IPA is a federation of Pompe disease patient groups worldwide that seeks to coordinate activities and share experience and knowledge among different international organizations.

Pompe Alliance

Pompe Alliance provides supportive services, education, and information to patients, caregivers, medical professionals, and Pompe community stakeholders.

Pompe Warrior Foundation (PWF)

The PWF promotes research, educates, and empowers individuals and families affected by Pompe disease and other lysosomal storage diseases.

United Pompe Foundation (UPF)

The UPF assists patients and/or their families with medical costs and other expenses they face and may not be able to cover, or fully cover, through their insurance.

Still have questions? Weโ€™ve got answers in our Frequently Asked Questions.

What important information should I know about safety?

POMBILITI in combination with OPFOLDA may cause serious side effects, including:

  • Hypersensitivity reactions (including anaphylaxis): Severe and potentially life-threatening allergic-type reactions related to the infusion have been reported during and after POMBILITI in combination with OPFOLDA treatment. Your doctor will inform you of the signs and symptoms of hypersensitivity reactions which may include: difficulty breathing or swallowing; rash or hives; low blood pressure; swelling of lips, tongue, throat, or face. Seek medical care immediately should signs and symptoms occur. If a severe reaction occurs, your doctor may decide to immediately discontinue the infusion and provide medical care. Appropriate medical support measures may be administered, and you may require close observation during and after POMBILITI infusion.
  • Infusion-Associated Reactions (IARs): Severe IARs related to the infusion have been reported during or after POMBILITI in combination with OPFOLDA. Your doctor will inform you of the signs and symptoms of hypersensitivity reactions which may include: hives, itching, shortness of breath, flushing, chills, and low blood pressure. Seek medical care immediately should signs and symptoms occur. If severe IARs occur during infusion, your doctor may decide to immediately discontinue the infusion and provide appropriate medical care. If you have an acute underlying illness at the time of POMBILITI infusion you may be at greater risk for IARs. If you have advanced Pompe disease you may have compromised heart and breathing function, which may put you at a higher risk of severe complications from IARs.
  • Risk of Acute Cardiorespiratory Failure: If you are likely to develop fluid volume overload or have an acute breathing condition or heart and/or breathing problems that require fluid restriction, you may be at risk of worsening of your heart or breathing status during POMBILITI infusion. Your doctor may decide that close observation during and after POMBILITI administration may be necessary.

Do not take POMBILITI in combination with OPFOLDA if you are pregnant.

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

What are POMBILITI + OPFOLDA?

POMBILITIยฎ (cipaglucosidase alfa-atga) + OPFOLDAยฎ (miglustat) are used in combination for treatment of adults with late-onset Pompe disease (LOPD) weighing 88 lbs or more who are not improving on their current enzyme replacement therapy (ERT).

It is not known if POMBILITI in combination with OPFOLDA is safe and effective in children with late-onset Pompe disease

Who is on my dedicated AMICUS ASSIST team?

Once youโ€™ve been prescribed POMBILITI + OPFOLDA and enrolled in AMICUS ASSIST, you will have access to a Patient Education Liaison (PEL) and a Case Manager. Your PEL can help you prepare for each treatment day, while your Case Manager will primarily assist you throughout many of the insurance-related aspects of your treatment.

How can I get help navigating insurance?

Your Case Manager can assist you in navigating your insurance coverage.

For more information about insurance, download the Insurance 101 brochure.

How can I get financial assistance?

We are committed to helping you access treatment. Your AMICUS ASSIST Case Manager can help identify possible sources of financial assistance, if necessary. Call AMICUS ASSIST for more information.

1-833-AMICUS-A ( 1-833-264-2872) Monday โ€“ Friday, 8AM โ€“ 8PM ET

What is a typical treatment day?

A typical treatment day should look like this:

Treatment day schedule
  • 2 hours before taking OPFOLDA:
    • Avoid all food and most beverages, except unsweetened beverages, such as water, tea, and/or coffee without cream, sugar, or other sweeteners. These you can drink as much as you want throughout the day
  • 1 hour before your POMBILITI infusion:
    • Take OPFOLDA exactly as directed by your healthcare provider, with an unsweetened beverage
  • 2 hours after taking OPFOLDA:
    • Resume normal eating and drinking

POMBILITI + OPFOLDA dosing is determined by your actual body weight and kidney function. Your healthcare provider will prescribe the dosage that is appropriate for you.

Treatment day schedule

Why do I have to fast while taking POMBILITI + OPFOLDA?

Certain medicines, including POMBILITI + OPFOLDA, require fasting because food and some drinks can affect the way these medicines work. In short, following the fasting rules helps your body absorb OPFOLDA, allowing it to stabilize POMBILITI so it can work the way it is supposed to.

If Iโ€™m already receiving another type of ERT, do I have to wait before I begin POMBILITI + OPFOLDA?

Thatโ€™s a great discussion to have with your healthcare provider. The good news is that when you are switching to POMBILITI + OPFOLDA, you can start POMBILITI + OPFOLDA on your next scheduled treatment day (in other words, 2 weeks after your last ERT dose).

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