Pronunciation:

se-be-LYE-pase AL-fa

Brand Names:

• Kanuma

Dosage Forms:

• Solution

Warnings:

Warning: Hypersensitivity Reactions Including AnaphylaxisPatients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.Initiate sebelipase alfa in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue sebelipase alfa and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur .

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Enzyme

Uses of This Medicine:

Sebelipase alfa injection is used to treat patients with Lysosomal acid lipase (LAL) deficiency.

This medicine is to be given 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 sebelipase alfa injection in children 1 month of age and older.

Older adults—

Appropriate studies on the relationship of age to the effects of sebelipase alfa injection have not been performed in the geriatric population, no geriatric-specific problems have been documented to date.

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:

• Allergy to eggs or egg products—May increase risk for allergic reactions to occur again.

Proper Use of This Medicine:

A nurse or other trained health professional will give you this medicine in a hospital. This medicine is given through an IV catheter that is placed into one of your veins. The medicine must be injected slowly, so the IV will need to stay in place for at least 2 hours. It is usually given once a week or once every other week.

Precautions While Using This Medicine:

Your doctor will check your progress closely while you are receiving this medicine. This will allow your doctor to see if the medicine is working properly and to decide if you should continue to receive it. Blood 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 or your child 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.

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

Anxiety or agitation

blurred vision

chest tightness

chills

coughing

diarrhea

difficulty with breathing or swallowing

dizziness

dry mouth

fever

headache

hives, itching, or rash

hoarseness

hyperventilation

irregular heartbeats

irritability

nausea

nervousness

pale skin

pounding in the ears

reddening of the skin, especially around the ears

restlessness

shaking

skin rash, encrusted, scaly, and oozing

slow or fast heartbeat

slow or irregular breathing

stomach pain

swelling of the eyes, face, inside of the nose, or throat

vomiting

trouble sleeping

unusual tiredness or weakness

Less common

Chest discomfort

fast, pounding, or irregular heartbeat or pulse

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

rapid shallow breathing

redness of the skin

redness of the white part of the eyes

swelling of the eyelids

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

Constipation

difficulty having a bowel movement (stool)

lack or loss of strength

muscle aches

pale skin

sneezing

sore throat

stuffy or runny nose

unusual bleeding or bruising

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.