Pombiliti: Uses, Side Effects and Dosage

Serious Warnings and Precautions

Hypersensitivity Reactions Including Anaphylaxis

• If you are treated with POMBILITI, you may experience a 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, infusion should be immediately paused, appropriate medical treatment should be initiated, and the benefits and risks of re-administration of POMBILITI should be considered.

Infusion-Associated Reactions (IARs)

• If you are treated with POMBILITI, you may experience severe IARs. If severe IARs occur, infusion should be immediately paused, appropriate medical treatment should be initiated, and the benefits and risks of re-administration of POMBILITI should be considered following severe IARs.

Risk of Acute Cardiorespiratory Failure in Susceptible Patients

• Individuals with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise due to fluid volume overload during POMBILITI infusion. Appropriate medical support and monitoring measures should be readily available during POMBILITI infusion.

What POMBILITI is used for:

• POMBILITI is a type of ‘enzyme-replacement therapy’ (ERT) that is used in combination with OPFOLDA (65 mg miglustat capsules) for the treatment of late--onset Pompe disease (LOPD) in adults weighing 40 kg or more.
• POMBILITI is always used with another medicine called 65 mg miglustat capsules. It is very important that you also read the Patient Medication Information of OPFOLDA (65 mg miglustat capsule).

How POMBILITI works:

People with Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA). This enzyme helps control levels of glycogen in the body.

In Pompe disease, high levels of glycogen build up in the muscles of the body. This keeps muscles, such as the muscles that help you walk, the muscles under the lungs that help you breathe, and the heart muscle, from working properly.

POMBILITI contains an artificial enzyme called cipaglucosidase alfa; it can replace the natural enzyme which is lacking in Pompe disease.

The ingredients in POMBILITI are:

Medicinal ingredients: cipaglucosidase alfa

Non-medicinal ingredients: citric acid monohydrate, mannitol, polysorbate 80, sodium citrate dihydrate

POMBILITI comes in the following dosage forms:

Lyophilized powder, 105 mg/vial

Do not use POMBILITI if:

• You have ever had life-threatening hypersensitivity or infusion-related reactions (e.g., a severe allergic reaction and severe skin reactions) to:

  • cipaglucosidase alfa
  • miglustat
  • any of the other ingredients of this medicine or its container
• A previous infusion had to be stopped and could not be restarted due to life-threatening hypersensitivity reactions or infusion-related reactions
• You are pregnant or planning to become pregnant.

To help avoid side effects and ensure proper use, talk to your healthcare professional before you take POMBILITI. Talk about any health conditions or problems you may have, including if you:

• have allergic reactions, including anaphylaxis (a severe allergic reaction) (see “Possible side effects from using POMBILITI” section for symptoms of life-threatening reactions).
• have an infusion-associated reaction while you are receiving the medicine or in the few hours afterwards (see “Possible side effects from using POMBILITI” section for symptoms of life-threatening reactions).
• have a history of heart or lung disease. These conditions may worsen during or immediately after your infusion with POMBILITI. Tell your healthcare professional or nurse immediately if you are experiencing shortness of breath, cough, rapid or irregular heartbeat, or any other effects from these conditions.
• if you have swelling in your legs or widespread swelling of your body, severe skin rash, or frothy urine when passing water. Your healthcare professional will decide if your POMBILITI infusion should stop, and they will give you appropriate medical treatment. Your healthcare professional will also decide if you can continue receiving POMBILITI.
• have kidney problems

Other warnings you should know about:

• Pre-treatment medications

  Your healthcare professional may give you other medicines before you have POMBILITI. These medicines include:

  • antihistamines and corticosteroids to prevent or help reduce infusion-related reactions.
  • antipyretics to reduce fever.

• Female patients

  Pregnancy and birth control

  • There is no experience with the use of POMBILITI in combination with 65 mg miglustat capsules during pregnancy.
  • You must not receive POMBILITI and/or take 65 mg miglustat capsules if you are pregnant. There may be risks to the unborn baby.

  • If you are able to become pregnant:

    • your healthcare professional will make sure you are not pregnant before you start taking POMBILITI in combination with 65 mg miglustat capsules.
    • you must use reliable birth control methods during use, and for 4 weeks after stopping both medicines.
    • tell your healthcare professional immediately if you get pregnant, think that you may be pregnant, or if you are planning to become pregnant.

Breast-feeding

• If you are breast-feeding, do NOT take POMBILITI in combination with 65 mg miglustat capsules. A decision will need to be made whether to stop treatment or to stop breast-feeding.
• Talk to your healthcare professional about the best way to feed your baby during treatment.
• Fertility – male and female patients
• Taking POMBILITI in combination with 65 mg miglustat capsules may affect your ability to have children. Talk to your healthcare professional if this is a concern for you.

• Driving and using machines

• Taking POMBILITI in combination with 65 mg miglustat capsules can cause dizziness, low blood pressure and drowsiness. You should use caution when driving or operating potentially dangerous machinery after receiving treatment or other pre-treatment medicines.

Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.

How to take POMBILITI:

POMBILITI will be given to you under the supervision of a healthcare professional. It is given through a drip into a vein. This is called an intravenous infusion.

You may be eligible for treatment at home based on evaluation by your healthcare professional. Your healthcare professional will decide upon evaluation if it is safe for you to have home infusion of

POMBILITI. If you get any side effects during an infusion of POMBILITI, your home infusion staff member may stop the infusion and start appropriate medical treatment.

POMBILITI should be used in conjunction with 65 mg miglustat capsules. You can only use miglustat 65 mg capsules with POMBILITI. Do NOT use miglustat 100 mg capsules (different product). Follow your healthcare professional’s instructions and read the Patient Medication Information of OPFOLDA (65 mg miglustat capsule) for their recommended dose.

Usual dose:

• The amount of medicine that you will be given is based on your weight. The recommended dose of POMBILITI is 20 mg for each kg of body weight once every other week as an intravenous infusion.

• 65 mg miglustat capsules are taken on the same day as POMBILITI. Refer to the Patient Medication Information of OPFOLDA (65 mg miglustat capsule) for information on how to take miglustat.

  • The POMBILITI infusion should start 1 hour after taking 65 mg miglustat capsules.
• In the event of a delay, the start of infusion should not exceed 3 hours from taking 65 mg miglustat capsules.
• The infusion of POMBILITI lasts approximately 4 hours.

Figure 1 - Dose timeline

* You should take 65 mg miglustat capsules 1 hour before receiving POMBILITI infusion. In the event of POMBILITI infusion delay, your healthcare professional will ensure that infusion is started within 3 hours of taking 65 miglustat capsules.

Switching from another enzyme replacement therapy (ERT):

If you are currently being treated with another ERT:

• Your healthcare professional will tell you when to stop the other ERT before starting POMBILITI.
• Tell your healthcare professional when you have completed your last dose.

Overdose:

There is no experience with overdose of POMBILITI and/or 65 mg miglustat capsules.

Missed Dose:

Forgotten dose:

If you miss a dose of 65 mg miglustat capsules, please speak to your healthcare professional and do not start your POMBILITI infusion. You will need to re-start the treatment of 65 mg miglustat capsules in combination with POMBILITI as soon as possible.

Infusion delays:

If you have missed an infusion and it has been more than 3 hours since you have taken 65 mg miglustat capsules, please contact your healthcare professional as soon as possible to reschedule your appointment. Your next appointment should be scheduled at least 24 hours after 65 mg miglustat capsules were last taken.

Possible side effects from using POMBILITI:

These are not all the possible side effects you may have when taking POMBILITI. If you experience any side effects not listed here, tell your healthcare professional.

POMBILITI is used with 65 mg miglustat capsules, and side effects can occur with either of these medicines. Side effects were mainly seen while patients were being infused with POMBILITI (infusion-related effects) or shortly after. You must tell your healthcare professional immediately if you get an infusion-associated reaction or an allergic reaction. Some of these reactions may become serious and life-threatening. Your healthcare professional may give you medicines before your infusion to prevent these reactions.

If you get any side effects during an infusion of POMBILITI, the infusion may be stopped, and appropriate medical treatment may be started.

The most significant infusion reactions included allergic reactions and allergic shock to POMBILITI.

Cardiorespiratory Risk

If you have underlying compromised cardiac or respiratory function (including respiratory illness), you may be at increased risk of acute cardiorespiratory failure from volume overload during POMBILITI infusion. Seek medical care if you experience acute onset of dizziness, shortness of breath, sweating, wheezing, leg swelling.

• Headache
• Involuntary shaking of one or more parts of the body
• Feeling sleepy
• Altered sense of taste
• Sensation like numbness, tingling, pins, and needles
• Rapid heartbeat
• Loose, runny stools
• Nausea
• Stomach pain
• Passing gas or wind
• Bloating
• Vomiting
• Trouble passing stools
• Increased sweating
• Muscle cramps, muscle pain, muscle weakness
• Pain in joints
• Feeling tired all the time
• Feeling uncomfortable in chest
• Swelling or pain in the body area where needle was inserted
• Pain
• Swelling in the hands, feet, ankles, legs
• Rise in blood pressure
• Stomach discomfort
• Cannot hold or maintain balance
• Burning sensation
• Pain in one or both sides of the head, throbbing pain, aura, eye pain, sensitivity to light (migraine)
• Unusual paleness of the skin
• Breathing difficult and triggers coughing, and shortness of breath (asthma)
• Throat discomfort
• Indigestion
• Sore or irritated throat
• Painful and abnormal contractions of the throat
• Mouth pain or discomfort (oral pain and oral discomfort)
• Swollen tongue
• Skin discoloration
• Swelling of the skin
• Pain in the area between the hip and rib
• Muscle fatigue
• Increased rigidity of muscles
• Constant feeling of being tired
• Pain in the cheek, gums, lips, chin
• Feeling jittery
• Feeling of uneasiness, overall feeling of being sluggish
• Pain in chest
• Swelling face
• Changes in body temperature
• Decrease in a type of white blood cell - shown in tests
• Scratch or damage to the skin

Serious side effects and what to do about them

If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

Reporting Side Effects

Storage:

Keep out of reach and sight of children.

Do not use this medicine after the expiry date which is stated on the bottle and carton after the letters “EXP”. The expiry date refers to the last date of that month.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines that you no longer use.

Unopened vials:

Do not freeze. Store under refrigeration (2°C to 8°C). Store in original container to protect from light.

After reconstitution and dilution:

After dilution, an immediate use is recommended.

The reconstituted product can be stored for up to 24 hours when refrigerated at 2°C to 8°C. Storage at room temperature is not recommended.

The diluted product can be stored for up to 24 hours when refrigerated at 2°C to 8°C, followed by up to 6 hours when stored at room temperature at 20°C to 25°C.

If you want more information about POMBILITI:

• Talk to your healthcare professional
• Find the full product monograph that is prepared for healthcare professionals and includes this Patient Medication Information by visiting the Health Canada Drug Product Database website: https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html, the manufacturer’s website: https://www.amicustherapeutics.ca, or by calling 1-833-810-5008.

Amicus Therapeutics Canada Inc.

100 King Street West, Suite 1600

1 First Canadian Place

Toronto ON M5X 1G5

Canada

Date of Authorization: 2025-04-15