Pronunciation:

ves-TRON-i-dase AL-fa - vjbk

Brand Names:

• Mepsevii

Dosage Forms:

• Solution

Warnings:

Anaphylaxis has occurred with vestronidase alfa-vjbk administration, as early as the first dose, therefore appropriate medical support should be readily available when vestronidase alfa-vjbk is administered. Closely observe patients during and for 60 minutes after vestronidase alfa-vjbk infusion. Immediately discontinue the vestronidase alfa-vjbk infusion if the patient experiences anaphylaxis .

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Vestronidase Alfa

Uses of This Medicine:

Vestronidase alfa-vjbk injection is used to treat Mucopolysaccharidosis VII (MPS VII) or Sly syndrome.

Sly syndrome is a disorder caused by the lack of an enzyme called beta-glucuronidase (GUS), which causes an abnormal build-up of toxic materials in the cells. Vestronidase alfa-vjbk acts by replacing the GUS enzyme and helps reduce the amount of toxic materials in the blood.

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 vestronidase alfa-vjbk injection in children. Safety and efficacy have been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of vestronidase alfa-vjbk 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.

Proper Use of This Medicine:

A nurse or other trained health professional will give you this medicine. It is given through a needle placed into one of your veins. It must be given slowly, so the needle will have to stay in place for at least 4 hours.

Your doctor may want you to stay around for at least 60 minutes after receiving this medicine to check for serious unwanted effects.

You may be given other medicines (eg, allergy medicine, fever medicine) 30 to 60 minutes before receiving this medicine to prevent unwanted effects.

Precautions While Using This Medicine:

It is very important that your doctor check your progress to make sure that this medicine is working properly and to check for unwanted effects.

This medicine may cause a serious allergic reaction, called anaphylaxis. This can be life-threatening and requires immediate medical attention. Tell your doctor right away if you have a rash, itching, hoarseness, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth after using 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:

Rare

Bloating or swelling of the face, arms, hands, lower legs, or feet

cough

difficulty swallowing

dizziness

fast heartbeat

hives, itching, skin rash

pale skin, pain, or redness at the injection site

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

rapid weight gain

tightness in the chest

tingling of the hands or feet

unusual tiredness or weakness

unusual weight gain or loss

Incidence not known

Fever with seizures

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:

Rare

Diarrhea

swelling at the injection site

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.