Pronunciation:

el-oh-SUL-fase AL-fa

Brand Names:

• Vimizim

Dosage Forms:

• Solution

Warnings:

Life-threatening anaphylactic reactions have occurred in some patients during elosulfase alfa infusions. Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms in conjunction with urticaria, have been reported to occur during infusions, regardless of duration of the course of treatment. Closely observe patients during and after elosulfase alfa administration and be prepared to manage anaphylaxis Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring .

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Enzyme

Uses of This Medicine:

Elosulfase alfa injection is used to treat patients with mucopolysaccharidosis type A (Morquio A syndrome).

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 elosulfase alfa injection in children. However, safety and efficacy have not been established in children younger than 5 years of age.

Older adults—

Although appropriate studies on the relationship of age to the effects of elosulfase 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:

• Fever or
• Lung or breathing problems (including sleep apnea)—Use with caution. May increase risk for more serious side effects.

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 at least 3.5 to 4.5 hours, once a week. You may also receive medicines to help prevent possible allergic reactions to the injection.

Precautions While Using This Medicine:

Your doctor will check your progress closely while you or your child 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.

This medicine may cause serious allergic reactions, including anaphylaxis. Anaphylaxis can be life-threatening and requires immediate medical attention. Call your doctor right away if you or your child have a rash, itching, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth while you are receiving this medicine.

Spinal or cervical cord compression (SCC) may occur while receiving this medicine. Check with your doctor right away if you or your child have back pain, loss of bladder or bowel control, or numbness or weakness in the legs.

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

Chills

cough

difficulty with swallowing

dizziness

fast heartbeat

fever

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

skin rash, hives, or itching

tightness in the chest

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

Abdominal or stomach pain

headache

vomiting

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.