Pronunciation:

peg-ue-ni-GAL-si-dase AL-fa - iwxj

Brand Names:

• Elfabrio

Dosage Forms:

• Solution

Warnings:

Warning: Hypersensitivity Reactions including AnaphylaxisPatients treated with pegunigalsidase alfa-iwxj have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during pegunigalsidase alfa-iwxj administration. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue pegunigalsidase alfa-iwxj immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to pegunigalsidase may be considered .

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Pegunigalsidase Alfa

Uses of This Medicine:

Pegunigalsidase alfa-iwxj injection is used to treat Fabry disease, which is an inherited disease caused by the lack of an enzyme called alpha-galactosidase A in the body. This enzyme is necessary for your body. Pegunigalsidase alfa-iwxj is used to help replace this enzyme.

This medicine is to be given only by or under the 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 pegunigalsidase alfa-iwxj injection in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of pegunigalsidase alfa-iwxj injection in geriatric patients.

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 problems—Use with caution. May be at a higher risk of severe complications from infusion reaction.

• Kidney disease—Use with caution. May make this condition worse.

Proper Use of This Medicine:

A nurse or other trained health professional will give you this medicine in a medical facility. This medicine is given through a needle placed in one of your veins. The medicine must be injected slowly, so your IV will need to stay in place for at least 1½ hours every 2 weeks.

Your doctor will give you other medicines (eg, allergy medicine, fever medicine, steroid) before starting treatment with this medicine.

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 while you are receiving this medicine to make sure that it is working properly. Blood and urine tests may be needed to check for unwanted effects.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with 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.

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 a fever, chills or shaking, chest pain, dizziness, flushing, headache, trouble breathing, hives, itching, or skin rash, lightheadedness, fainting, fast, pounding, or uneven heartbeat, or trouble breathing after receiving this medicine.

Check with your doctor right away if you have cloudy or bloody urine, high blood pressure, or swelling of the face, feet, or lower legs. These maybe symptoms of serious kidney problem.

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:

More common

Back pain

bloody or cloudy urine

burning, numbness, tingling, or painful sensations

chest tightness

chills

cough

ear congestion

fast heartbeat

fever

flushing

headache

loss of voice

muscle aches

nausea and vomiting

nerve pain

pain in the arms, legs, lower back, bottom, or hips

skin itching, rash, or redness

sneezing

sore throat

stuffy or runny nose

swelling of the face, throat, or tongue

trouble breathing

trouble swallowing

unsteadiness or awkwardness

unusual tiredness or weakness

weakness in the arms, hands, legs, or feet

Incidence unknown

Blurred vision

chest pain or discomfort

confusion

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

nervousness

pounding in the ears

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

shakiness in the legs, arms, hands, or feet

slow or irregular heartbeat

sweating

swelling of the face, feet, or lower legs

trembling or shaking of the hands or feet

vomiting

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

pain or tenderness around the eyes and cheekbones

stomach pain

Incidence not known

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

difficulty in moving

joint pain

lack or loss of strength

muscle cramps, pains, or stiffness

sneezing

swollen joints

throat irritation

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.