Elosulfase Alfa (Intravenous)

Elosulfase alfa is a generic medicine solution used to treat patients who have a rare metabolic disorder known as Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

Overview

Elosulfase alfa is given as a constant infusion through a vein (intravenous)of the patient. It is usually given at a hospital or clinic, under the direct supervision of a doctor. Each infusion therapy can last about three to five hours.

Morquio A syndrome is a rare, metabolic disorder caused when the body does not make enough of a special enzyme needed to break down materials the body cannot use. This results in a buildup of waste that leads to other medical conditions, such as skeletal deformities.

When infused into the body, the medicine replaces the deficient enzyme. It then aids in the breakdown and removal of unwanted materials from the body.

Elosulfase alfa is safe and effective for treating adults and certain children. Since it is not a cure for the condition it treats, patients may have to take the treatment for the rest of their lives.

It may cause a serious, possibly life-threatening allergic reaction called, anaphylaxis. Spinal or cervical cord compression (SCC) is another serious condition that has occurred from using this drug.

This intravenous (IV) medicine is sold in the US under the brand name, Vimizim, and is intended for use by hospitals and clinics.

Condition treated

  • Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)

Type Of Medicine

Hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme

You should consider the potential benefits and risks of using elosulfase alfa injection. Your doctor may help you decide if it is safe for you, and determine how to treat you.

In discussing your medical history, tell your doctor if you have had an unusual or allergic reaction to this or other medicines. Some patients may also be allergic to inactive ingredients in medicines.

Also let your doctor know if you have any allergy to foods, preservatives, dyes or animals.

Certain medical conditions may affect the way the injection works. tell your doctor if you have any other medical problems, especially if you have sleep apnea, fever or an infection.

Give your doctor a list of all medications you are taking or plan to take. These include prescription and over-the-counter medicines (OTC's), vitamins, nutritional supplements, and herbal products.

If you are within the following groups, your doctor will determine if (and how) to treat you, based on the potential benefits and risks involved.

Pediatrics

The medicine is proven safe and effective in patients five years of age and older. Your child's doctor may still need to take precautions when treating your child.

Geriatrics

There are no specific problems found that would limit the use of the medicine in elderly patients.

Pregnancy

Tell your doctor if you are pregnant, may be pregnant or plan to become pregnant. Adequate studies have not been done to determine risks posed to an unborn child.

Breastfeeding

There is no specific information available that suggests the medicine can pass through breast milk and harm a breastfeeding infant.

Side Effects

Side effects, hypersensitivity, or allergic reactions may occur from taking the injection. Your doctor will monitor you closely during the IV infusion. The doctor will be able to determine whether the medicine is working as it should.

Before infusion begins, you may be given medications such as antihistamines which may help reduce the risk of side effects.

Serious allergic reaction

A serious allergic reaction known as, anaphylaxis, may occur. This life-threatening reaction can happen during or after infusion (up to three hours later). Hypersensitivity reactions have occurred up to six days later.

Symptoms of a serious allergic reaction (anaphylaxis) are as follows. Call your doctor right away if they occur:

  • Shortness of breath
  • Hives
  • Itching
  • Flushing
  • Cough

If these symptoms occur, your doctor, nurse or other healthcare professional should slow down the infusion or stop it altogether. You may be given additional medication to relieve the reaction.

The infusion should be stopped immediately, and you should be treated if you experience a severe reaction.

Serious side effect

A serious side effect known as spinal or cervical cord compression (SCC) may occur during infusion of the medicine. Call your doctor immediately if you experience the following symptoms:

  • Back pain
  • Weakness or numbness in the legs
  • Loss of bladder or bowel control

The following side effects may also occur and need immediate medical attention. Call your doctor right away if you experience any of them.

More commonly occur

  • Fast heartbeat
  • Dizziness
  • Chills
  • Fever
  • Cough
  • Tightness in the chest
  • Difficulty swallowing
  • Hives, itching or skin rash
  • Unusual weakness or tiredness
  • Swelling or puffiness of the eyelids or around the eyes, lips, face or tongue

Some side effects which may occur tend to go away on their own as the body gets used to the medicine. They may require medical attention only if they get worse, become bothersome or do not go away. Tell your doctor if you have the following side effects:

More commonly occur

  • Vomiting
  • Stomach or abdominal pain
  • Headache

The lists provided do not include all possible side effects. Check with your doctor if you notice any unusual symptom that becomes bothersome.

You can ask your doctor or health care professional about ways to prevent or reduce side effects. You may also call the FDA at 1-800-FDA-1088 to report side effects.

Dosage

The injection is supplied to hospitals and clinics as single-use vials of 5 mg/5 mL (1 mg/mL) elosulfase alfa solution. The injection will be given into one of your veins by a nurse of healthcare professional, usually under the supervision of a doctor.

Adults and children 5 years and older

The medicine should be slowly infused into your body through an IV tube. A treatment can take about 3 to 5 hours and needs to be done once a week.

Your doctor may give you medicines, such as antihistamines, about 30 to 60 minutes before infusion begins. This may help prevent allergic reactions to the injection.

Missed dose

If you missed your appointment for infusion, call your doctor or nurse right away to reschedule your treatment session.

Overdose

Overdose of the medicine is unlikely since a doctor usually supervises the infusion process.

Interactions

Food, alcohol, and tobacco

Your doctor or healthcare professional may tell you whether you should avoid certain foods, alcohol or tobacco during treatment with the injection.

Other medicines

To date, there is no known medicine that causes adverse interaction with the injection. This does not mean that the injection cannot interact with other drugs.

Your doctor can take the necessary precautions once you disclose the list of all medications you use or plan to use during your treatment.

Other medical problems

Other medical conditions may affect the way the medicine works. Patients should let their doctor know if they have any other medical problem, especially the following:

  • Lung or breathing problems (including sleep apnea)
  • Fever
  • Infection

The medicine should be used with caution in patients with those conditions as it has the potential to increase the risk for more serious side effects.

Warnings

  • Do not use this medicine to treat your child if the child is under the age of five. Where treatment is necessary, it must be determined by your child's doctor.
  • The medicine is given to children based on weight. Tell your doctor if your child weight changes.
  • You must call your doctor right away if you develop an allergic reaction, any of the side effects listed, or any unusual symptoms that is bothersome. They may be serious and require urgent medical attention.
  • Ensure you monitor your health closely when you are away from the hospital. Hypersensitivity reactions may occur up to six days after receiving the treatment and may need medical attention.

Storage

Elosulfase alfa injection is given at a hospital or clinic and is usually stored there.

Summary

Elosulfase alfa's approval by the US Food and Drug Administration (FDA) provided a safe and effective treatment for patients with the rare condition, Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

The injection is safe and effective for use in adults and children 5 years of age and older. In addition to children younger than 5 years of age, the drug may not be safe to use in patients with certain medical conditions.

Unlike other medicines, there is no known medicine, or substance, that cause adverse interaction with elosulfase alfa.

There are few known side effects, some of which do not commonly occur.

However, the medicine may cause a serious allergic reaction known as anaphylaxis. It may also cause spinal or cervical cord compression (SCC). These conditions require urgent medical attention.

Patients may have to take elosulfase alfa injection for the rest of their lives. While the medicine may work well for enzyme replacement, it cannot cure patients with morquio A syndrome.