Elosulfase alfa

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

Elosulfase alfa is used to treat some symptoms of a genetic condition called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis) IV (4) type A, or MPS IV A, which is also known as Morquio A syndrome.

MPS IVA 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.

Elosulfase alfa may help improve walking and stair-climbing abilities in people with this condition. However, this medication is not a cure for MPS IVA.

Elosulfase alfa may also be used for other reasons not mentioned in this medication guide.

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

Elosulfase alfa must be given slowly, and the infusion can take up to 4 hours or longer to complete.

Elosulfase alfa is usually given once per week. Follow your doctor's instructions.

Elosulfase alfa doses are based on weight (especially in children and teenagers). Your dose needs may change if you gain or lose weight.

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

You will be watched closely after receiving elosulfase alfa, to make sure you do not have an allergic reaction.

An allergic reaction may happen during or shortly after you receive this medicine. Tell your caregivers or get emergency medical help immediately if you notice any signs of a severe allergic reaction, such as chest discomfort, cough, trouble breathing, vomiting, skin rash or redness, pale skin, blue lips or fingernails, and feeling faint.

You might be at a higher risk of having a reaction to elosulfase alfa if you have a breathing disorder or if you have a fever when you get your infusion.

You will be monitored closely after receiving elosulfase alfa to ensure you do not have an allergic reaction.

Your elosulfase alfa infusion may be delayed if you have a fever or cold symptoms.

Tell your doctor if you have ever had:

• breathing problems; or
• sleep apnea, and you use a continuous positive airway pressure (CPAP) machine.

Tell your doctor if you are pregnant or breastfeeding.

It is not known whether elosulfase alfa will harm an unborn baby. However, MPS symptoms can get worse during pregnancy and may cause medical problems in both mother and baby. The benefit of treating MPS may outweigh any risks to the baby.

Your name may need to be listed on a Morquio A Registry while you are using elosulfase alfa. The purpose of this registry is to track the progression of this disorder and the effects that elosulfase alfa has on long-term treatment of Morquio A syndrome. The registry also tracks the effects of elosulfase alfa on a baby 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 shortly after the infusion of elosulfase alfa (up to 3 hours later). Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as:

• feeling faint, even while lying down;
• cough, chest discomfort, feeling short of breath;
• skin rash or redness, sudden warmth or tingling;
• nausea, vomiting;
• pale skin; or
• blue lips or fingernails.

Common side effects of elosulfase alfa may include:

• nausea, vomiting, stomach pain;
• headache;
• fever, chills; or
• feeling tired.

Even though it may not be a side effect of elosulfase alfa, increased pressure on the spinal cord is a complication of MPS IVA that may happen while you are using this medicine. Tell your doctor right away if you have any symptoms of spinal cord compression:

• back pain;
• loss of movement in any part of your body; or
• loss of bowel or bladder control.

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 IV:

2 mg/kg via IV infusion once a weekComments:-Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.-See Other Comments/Administration Advice for recommended dilution volume and infusion rates.Use: For patients with mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome)

Usual Pediatric Dose for Mucopolysaccharidosis Type IV:

5 years or older: 2 mg/kg via IV infusion once a weekComments:-Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.-See Other Comments/Administration Advice for recommended dilution volume and infusion rates.Use: For patients with mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome)

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

Since this medication is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

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

Other drugs may affect elosulfase 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.