Pronunciation:

sip-a-gloo-KOE-si-dase AL-fa - atga

Brand Names:

• Pombiliti

Dosage Forms:

• Powder for Solution

Warnings:

Severe Hypersensitivity Reactions, Infusion-Associated Reactions, and Risk of Acute Cardiorespiratory Failure in Susceptible PatientsHypersensitivity Reactions Including AnaphylaxisPatients treated with cipaglucosidase alfa-atga have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during cipaglucosidase alfa-atga administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, cipaglucosidase alfa-atga should be discontinued immediately, and appropriate medical treatment should be initiated. In patients with severe hypersensitivity reaction, desensitization measures to cipaglucosidase alfa-atga may be considered.Infusion-Associated Reactions (IARs)Patients treated with cipaglucosidase alfa-atga have experienced severe IARs. If severe IARs occur, immediately discontinue the cipaglucosidase alfa-atga infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering cipaglucosidase alfa-atga following severe IARs. Patients with an acute underlying illness at the time of cipaglucosidase alfa-atga 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 PatientsPatients 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 cipaglucosidase alfa-atga infusion. More frequent monitoring of vitals should be performed during cipaglucosidase alfa-atga infusion in such patients .

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Cipaglucosidase Alfa

Uses of This Medicine:

Cipaglucosidase alfa-atga injection is used in combination with miglustat to treat late-onset Pompe disease in patients who are not improving on their current enzyme replacement therapy (ERT). It contains a human enzyme called acid alpha-glucosidase, which helps with the digestion and absorption of glycogen. People with Pompe disease are not able to make enough of this enzyme.

This medicine is to be given only by or under the direct supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of cipaglucosidase alfa-atga injection in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of cipaglucosidase alfa-atga injection in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Heart disease or
• Lung or breathing problems—Use with caution. May make these conditions worse.

Proper Use of This Medicine:

A nurse or other trained health professional will give you or your child this medicine in a hospital. This medicine is given through a needle placed in one of your veins. The medicine must be injected slowly, so your IV tube will need to stay in place for 4 hours every other week.

Your doctor will give you other medicines (eg, allergy, fever, or steroid medicine) before you receive this medicine to help prevent unwanted effects.

Missed dose—

Call your doctor or pharmacist for instructions.

Precautions While Using This Medicine:

It is very important that your doctor check your progress closely to make sure this medicine is working properly and to decide if you should continue to receive it. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant with can harm your unborn baby. If you are woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before receiving this medicine. Use an effective form of birth control during treatment and for at least 60 days after the last dose. If you think you have become pregnant whiles using this medicine, tell your doctor right away.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, skin rash, trouble breathing, or unusual tiredness or weakness after receiving this medicine.

This medicine may cause infusion-related reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you start to have chest discomfort, fever, chills or shaking, confusion, difficulty swallowing, dizziness, fast heartbeat, flushing, redness of the skin, increase or decrease blood pressure, trouble breathing, itching or rash, unusually warm skin, or lightheadedness, or fainting after receiving this medicine.

Talk with your doctor before using this medicine if you plan to have children. Some men and women who use this medicine have become infertile (unable to have children).

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

Less common

Blurred vision

chest tightness

chills

confusion

cough

difficulty swallowing

dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position

fast, pounding, or irregular heartbeat or pulse

feeling of warmth

fever

headache

hives, itching, skin rash

increased blood pressure

lower back or side pain

nausea and vomiting

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

redness of the face, neck, arms and occasionally, upper chest

shakiness in the legs, arms, hands, or feet

sweating

trembling or shaking of the hands or feet

trouble breathing

unusual tiredness or weakness

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Diarrhea

Less common

Acid or sour stomach

belching

burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings

change in taste

constipation

difficulty in moving

full or bloated feeling

general feeling of discomfort or illness

heartburn

indigestion

joint or muscle pain

lack or loss of strength

loss of taste

muscle spasms

pressure in the stomach

stomach discomfort, upset, or pain

swelling of the stomach area

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.