Pronunciation:

oh-LIP-ue-dase AL-fa - rpcp

Brand Names:

• Xenpozyme

Dosage Forms:

• Powder for Solution

Warnings:

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

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Olipudase Alfa

Uses of This Medicine:

Olipudase alfa-rpcp injection is used to treat non-central nervous system symptoms of acid sphingomyelinase deficiency (ASMD). ASMD is a genetic disease that affects many organs (eg, liver, lungs, spleen, blood, stomach) and can worsen over time.

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 performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of olipudase alfa-rpcp injection in children. Safety and efficacy have been established.

Older adults—

Appropriate studies on the relationship of age to the effects of olipudase alfa-rpcp injection have not been performed in the geriatric population.

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.

Proper Use of This Medicine:

A doctor or other trained health professional will give you or your child this medicine in a medical facility. It is given through a needle that is placed into one of your veins.

This medicine must be given slowly, so the needle will have to remain in place for at least 20 minutes. It is usually given every 2 weeks.

You may also receive medicines (eg, allergy medicine, fever medicine, steroids) to help prevent possible unwanted effects during the injection.

Missed dose—

Call your doctor or pharmacist for instructions.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can get pregnant, your doctor may give you a pregnancy test before you start using this medicine to make sure you are not pregnant. Use an effective form of birth control during treatment with this medicine and for at least 14 days after the last dose. If you think you have become pregnant while using this medicine, tell your doctor right away.

This medicine may cause serious allergic reactions, including anaphylaxis and angioedema, 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, or skin rash, large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, 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, dizziness, trouble breathing, itching or rash, lightheadedness or fainting after receiving this medicine.

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

Blurred vision

body aches or pain

chest tightness

confusion

cough

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

dryness or soreness of the throat

fast, pounding, or irregular heartbeat or pulse

fever

hives, itching, or skin rash

hoarseness

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

stuffy or runny nose

sweating

tender, swollen glands in the neck

trouble breathing

trouble in swallowing

unusual tiredness or weakness

voice changes

Incidence not known

Back pain

chills

headache

large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or sex organs

nausea and 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

difficulty in moving

flushing, redness of the skin

lack or loss of strength

muscle or joint pain

redness of the eye

sneezing

throat irritation

unusually warm skin

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.