Velaglucerase Alfa (Intravenous)

Velaglucerase alfa is an enzyme replacement medication used in the treatment of type 1 Gaucher's disease in order to allow the body to process fats correctly. It is taken by intravenous solution every two weeks.


Velaglucerase alfa is a medication used in the treatment of type 1 Gaucher's disease. Type 1 Gaucher's disease is caused when the body does not produce glucocerebrosidase, a naturally occurring enzyme that processes fats in the body correctly. If left untreated, fat will build up in the liver, kidneys, spleen, brain, lungs, and bone marrow, causing dangerous organ enlargement, anemia and bruising, fatigue, and a low blood platelet count. Velaglucerase alfa is a type of enzyme that has an identical amino acid sequence to glucocerebrosidase, meaning that it can work the same way that glucocerebrosidase does to process fats in the body.

Velaglucerase alfa is available in individual vials as a powder solution for use in a liquid solution, and is to be taken intravenously under a physician's supervision every two weeks. It can cause mild side effects, such as unusual sensation or rashes on the skin. More serious side effects include flu-like symptoms, respiratory symptoms, or irregularities in blood pressure.

Conditions treated

  • Type 1 Gaucher's Disease

Type of Medicine

  • Enzyme Replacement Therapy

Side Effects

Mild Side Effects

Velaglucerase alfa can cause some unwanted side effects in patients. Some side effects are mainly unpleasant, and are not necessarily cause for serious medical concern. These side effects are caused by your body adjusting to the medicine and often subside after a period of time. Common side effects may include unusual back pain or joint pain, a feeling of weakness or loss of strength, and mild abdominal or stomach pain. Less common side effects involve skin irritation, including symptoms such as raised welts or hives on the skin, a redness of the skin near the face, neck, chest, arms, or upper chest, and a feeling of warmth or itchiness on the skin. These side effects should be reported to your physician, who may be able to help you with a course of treatment to relieve your symptoms.

Serious Side Effects

Velaglucerase alfa may cause side effects that require medical attention. Common side effects like this include fever or chills, nausea or vomiting, headaches or body aches. You may experience an unusual skin rash, congestion of the ear, or facial swelling. Seek medical attention immediately if you experience respiratory symptoms like a sore throat sometimes accompanied by laryngitis, nasal congestion or a runny nose, coughs, sneezing, shortness of breath, unusual weakness or a feeling of tiredness, or dizziness.

Some less common side effects that still require medical attention include bone pain or blurred vision. Look out for dizziness, faintness, or a lightheaded feeling when moving or getting up, as well as persistent confusion. You may experience a heartbeat that feels unusually slow or fast, pounding, or irregular, sometimes accompanied by nervousness. This may include a pounding in the ears. If you experience any of these symptoms, seek medical intervention immediately.


The dosages discussed here for velaglucerase alfa, including the intervals of dosage and the amounts in a dosage, are standard guidelines recommended by the manufacturer. Your doctor may prescribe a different dosage for the medication depending on your medical condition and other health factors. Always refer to the instructions provided by your doctor when taking medication.

Velaglucerse alfa is available as a powder that can be made into a solution. It is administered intravenously as a 60-minute IV infusion, and will need to be done under the supervision of a qualified healthcare professional every two weeks. The standard dosage per session is 60 units per kilogram for both adults and children older than 4 years of age.

Major Drug Interactions

Like any medication, velaglucerase alfa can have reactions with other drugs and substances that are in the body. These reactions can sometimes negate, exaggerate, or blunt the effects of either medication, or can trigger unwanted side effects. Patients taking velaglucerase alfa should keep a complete list of all medications they are currently taking, whether the medications are prescription or over the counter. A doctor or pharmacist will be able to help you identify potential interactions with velaglucerase alfa and your other medications.

If you experience an unexpected side effect or new symptom of velaglucerase alfa when adding a new drug, medication, or over-the-counter supplement to your regimen, inform your doctor as soon as possible. Not all side effects or interactions are cause to discontinue a medication. Your doctor will help you determine what precautions to take about your side effects or drug interactions, and may want to change your dosage.

Using alcohol or tobacco with velaglucerase alfa can cause unwanted interactions to occur. Talk to your doctor about whether it's safe to partake of these substances while you are under a regimen of velaglucerase alfa.


Hypersensitivity Reaction

Velaglucerase alfa is an intravenous protein product, and may cause a hypersensitivity reaction in some individuals. These symptoms are usually mild and do not often pose a serious threat to health, but rather some discomfort and inconvenience. These symptoms occur most often in the first 6 months of a velaglucerase alfa regimen, and normally subside over time.

The symptoms of a hypersensitivity reaction to velaglucerase alfa include hypertension (very high blood pressure) or hypotension (very low blood pressure), in addition to dizziness and headaches, physical muscle weakness and general fatigue, and a feeling of warmth or feverishness. You may also experience some discomfort in the chest area, unusual itching on the skin, and shortness of breath or unusually labored breathing.

In case of a hypersensitivity reaction to velaglucerase alfa, contact your physician. Hypersensitivity response is not necessarily cause to discontinue a velaglucerase regimen. Depending on the nature of the reaction and the number of your symptoms, your doctor may decide to slow your infusion rate, or may decide to halt and then restart your regimen with a different infusion time. Your doctor may also add medications for your symptoms into your regimen, such as steroid creams or antihistamines.

Anaphylactic Reaction

Velaglucerase alfa, like all substances, comes with some risk of an allergic or anaphylactic reaction. Symptoms can range from mild to life-threatening, and should be treated immediately. If you experience allergic or anaphylactic reactions that are not listed as side effects of velaglucerase alfa, or your side effects suddenly become worse, get medical help immediately to combat the anaphylactic reaction. A single anaphylactic reaction may be mild, but subsequent reactions can be stronger and can even lead to life-threatening anaphylactic shock.

Maternal Risk

Pregnant individuals with type 1 Gaucher's disease, which velaglucerase alfa is used to treat, have a higher risk of spontaneous abortion and com/health/miscarriage/">miscarriage than average. During pregnancy, the symptoms of type 1 Gaucher's disease may worsen or may manifest with symptoms not previously experienced by the pregnant patient.

Animal studies have not shown an increased risk of bodily harm to the fetus or carrier of the fetus while taking velaglucerase alfa. However, this effect has not yet been tested sufficiently in pregnant humans with type 1 Gaucher's disease to determine whether velaglucerase alfa is a threat to the life of parent or fetus when used during a pregnancy. Therefore, caution is recommended when using this medication during a pregnancy, and should only be used if clearly needed for management of the patient's symptoms.



Velaglucerase alfa comes individually packed in glass vials. These vials are closed with a rubber stopper, an aluminium seal, and a plastic cap which can be flipped off to access the medication inside. The medication itself comes as a powder, and needs to be diluted prior to use. Each vial is designed for a single use only, and contains either 200 units or 400 units. Your physician will know how to dilute and prepare velaglucerase alfa.

Conditions for Storage

Velaglucerase alfa should be stored inside of a refrigerator at 36ºF to 46ºF, or at 2 to 8ºC. Do not store this medication in the freezer or expose it to temperatures below freezing. Protect this medication from light.

Expired Medication

If your velaglucerase alfa has expired, do not use it. Your pharmacist may have a drug disposal or take-back program. If not, dispose of your velaglucerase alfa immediately and safely.


Velaglucerase alfa is used to treat type 1 Gaucher's disease by replacing the missing enzyme that lets your body process fat correctly.

Velaglucerase alfa comes in vials as a powder, and is diluted to form an intravenous solution. This is normally administered every two weeks by a trained healthcare professional.

Mild side effects from velaglucerase alfa include back or joint pain, physical weakness, and stomach or abdominal pain, rashes, and flushed skin. These are not necessarily symptoms of anything serious and often go away after your body gets used to the medication. More serious side effects include flu-like symptoms such as a fever, nausea, headaches and body aches, or skin rashes. You may also experience symptoms of a cold, sore throat, or laryngitis, as well as persistent tiredness, dizziness, or confusion. Hypertension or hypotension may also occur. These are all reasons to seek medical attention immediately.

Last Reviewed:
December 26, 2017
Last Updated:
April 05, 2018
Content Source: