Vestronidase alfa

Vestronidase alfa contains an enzyme that is normally found in the body of healthy individuals. Some people do not have this enzyme due to a genetic disorder. Vestronidase alfa helps provide this missing enzyme for those individuals.

Vestronidase alfa is used to treat some symptoms of a genetic condition known as mucopolysaccharidosis VII (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis Seven) or MPS VII, which is also referred to as Sly syndrome.

MPS VII is a metabolic disorder where the body does not have the enzyme needed to break down certain natural substances. These substances can accumulate in the body, leading to enlarged organs, abnormal bone structure, changes in facial features, breathing difficulties, heart issues, vision or hearing loss, and changes in mental or physical abilities.

Vestronidase alfa may help improve walking ability in individuals with this condition. However, this medication is not a cure for MPS VII.

Vestronidase alfa may also be used for other purposes that are not mentioned in this medication guide.

Vestronidase alfa is given as an infusion into a vein. A healthcare provider will give you this injection.

vestronidase alfa is usually given once every 2 weeks. Vestronidase alfa must be given slowly, and the infusion can take about 4 hours to complete.

About 30 to 60 minutes before each injection, you will be given other medications to help prevent a serious allergic reaction.

Tell your caregivers if you feel any burning, pain, or swelling around the IV needle when vestronidase alfa is injected.

You will be watched closely for at least 60 minutes after receiving vestronidase alfa, to make sure you do not have an allergic reaction.

Vestronidase alfa doses are based on weight. Your dose needs may change if you gain or lose weight.

An allergic reaction may happen during or shortly after the infusion of this medicine. Tell your caregivers or seek emergency medical help immediately if you notice any signs of a severe allergic reaction, such as sweating, difficulty breathing, pale skin, blue lips or fingernails, and feeling faint.

Tell your doctor if you have ever had:

• a lung disease or breathing problems.

Tell your doctor if you are pregnant or breastfeeding.

Get emergency medical help if you have signs of an allergic reaction: hives; difficulty breathing; swelling of your face, lips, tongue, or throat.

An allergic reaction may happen during or soon after the infusion of vestronidase alfa. Tell your caregivers or get emergency medical help right away if you notice any signs of a severe allergic reaction, such as:

• feeling faint, even while lying down;
• sweating, cold or clammy skin;
• feeling short of breath;
• pale skin; or
• blue lips or fingernails.

Common side effects of vestronidase alfa may include:

• diarrhea;
• swelling in your hands or feet;
• rash, itching; or
• swelling or irritation around the IV needle.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Adult Dose for Mucopolysaccharidosis Type VII:

4 mg/kg IV infusion once every 2 weeksUse: For the treatment of Mucopolysaccharidosis VII (MPS VII) also known as Sly syndrome

Usual Pediatric Dose for Mucopolysaccharidosis Type VII:

4 mg/kg IV infusion once every 2 weeksUse: For the treatment of Mucopolysaccharidosis VII (MPS VII) also known as Sly syndrome

Call your doctor for instructions if you miss an appointment for your Vestronidase alfa injection.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Other drugs may affect vestronidase alfa, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.