Agalsidase Beta (Intravenous)

Beta is a drug used in the treatment of Fabry disease which is a genetic condition that prevents the production of alpha-galactosidase A, a necessary enzyme.

Overview

Fabry disease is a genetic condition that prevents the production of the necessary enzyme, alpha-galactosidase A. The drug is administered intravenously every two weeks with a dosage that equals 1mg for every 1kg of body weight. This drug is used to treat the symptoms of Fabry disease such as gastrointestinal problems, hearing loss, mitral valve prolapse, and problems with the kidneys accompanied by pain in that area.

The disease affects the X chromosome and is more prevalent in males, with very few known cases in female patients. In the United States, 1 out of every 40,000 males may be affected. Along with treatment to replace the missing enzyme, Fabry disease can affect the kidneys and transplantation can be necessary in some cases. The life expectancy for males afflicted with the disease is approximately 58 years old, and in the rare instances when Fabry's affects women, their life expectancy is over 75 years old.

The lack of the alpha-galactosidase A enzyme can cause many problems in the body, especially to the kidneys. The treatment of the disease with Agalsidase Beta must be conducted under close medical supervision and at a full-service treatment facility. The likelihood of a reaction to the infusion can be lessened with the use of certain drugs designed for that specific treatment purpose. Physicians that treat Fabry disease can offer the patient advice and steps to follow to reduce side effects of the medicine.

Conditions Treated

  • Fabry disease

Type of Medicine

  • Endocrine-Metabolic Agent

Side Effects

In addition to the side effects of the medicine, Fabry disease can cause significant damage to the kidneys and patients suffering from the condition will need to receive consistent treatment from a nephrologist to monitor their condition. The side effects of any medications that the patient is taking must be monitored due to the damage that the disease causes to the patient's kidneys.

If the patient begins to experience any side effects the prescribing physician should be notified immediately. The most common side effects of Agalsidase Beta include:

  • blurred vision
  • chest pain or discomfort
  • confusion
  • a cough producing mucus
  • difficulty breathing
  • difficulty in moving
  • dizziness
  • fatigue
  • faintness or lightheadedness when getting up from a lying or sitting position suddenly
  • feeling unusually cold combined with shivering
  • headache
  • hives or welts
  • irregular heartbeat
  • itching
  • joint pain
  • muscle aching or cramping
  • muscle pains or stiffness
  • nervousness
  • pounding in the ears
  • redness of the skin
  • shortness of breath
  • skin rash
  • stomach pain
  • sweating
  • swelling of the ankles, feet, and lower legs
  • swollen joints
  • tightness in the chest
  • an unusual feeling of tiredness or weakness
  • wheezing

There may be practices that can lessen or prevent the appearance of side effects, and these steps should be discussed with the medical team prior to infusion. Many of these side effects are a result of the body adjusting to the infusion and will often go away on their own. It is best to always notify the physician handling the case of any instances of side effects, regardless of their severity.

Other side effects that can occur due to the infusion include:

  • acid or sour stomach
  • belching
  • body aches or pain

The body produces a substance that can bind to the drug making it less effective or cause side effects, including: burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings, congestion, discouragement, dryness or soreness of the throat, fear, feeling sad or empty, fever, heartburn, hoarseness, indigestion, irritability, lack of appetite, loss of interest or pleasure, nausea, pain or tenderness around eyes and cheekbones, paleness of skin, a runny nose, the sensation of change in temperature, skeletal pain, sneezing, stuffy nose, swelling of the testes, tender or swollen glands in neck, trouble concentrating, trouble sleeping, trouble in swallowing, and voice changes.

Due to the emotional nature of some of these side effects, it is important to keep in touch with the medical team following the infusion and report any side effects regardless of their severity.

Dosage

The dosage requirements of Agalsidase Beta differ for every patient, therefore it is imperative to closely follow your doctor's orders when taking this drug. Typically, the drug is administered every two weeks intravenously. The dosage amount is determined by the physician and is reliant on the patient's body weight and other factors.

1mg per 1kg of body weight is the standard, although it is important to remember that every case is different. The dosage is injected directly into the patient's vein and can be repeated every two weeks. Because this drug has not been sufficiently studied for use in children, it is important to consult with a physician to determine if it is an appropriate treatment option, and what dosage is effective for the child.

Agalsidase Beta has only been studied for use in adults, and there is no data available on how this drug affects pediatric patients. Additionally, side effects from the use of the drug during pregnancy or when breastfeeding are not extensive but the limited studies did not reveal any negative side effects. With this lack of conclusive data, it is best that every patient review potential side effects with their physician if they are thinking of using Agalsidase while pregnant or breastfeeding.

If a scheduled dosage is not administered at the prescribed time, it is important to call the patient's doctor or pharmacist immediately.

Major Drug Interactions

The infusion process that is used to administer Agalsidase Beta can create negative interactions with patients being treated for heart conditions. The most important area of concern for patients receiving this drug is the need of a complementary drug that will protect from the side effects that are inherent with infusions. This drug should only be administered in a full-service treatment facility under close medical supervision.

Warnings

A complementary medicine that will protect the patient from infusion reactions and possible infection must also be taken during the use of Agalsidase Beta. The drug should always be administered in a healthcare facility that is equipped to treat any infusion reactions that may occur.

Similar to other drugs that are administered via infusion, Agalsidase Beta must be given under close medical supervision and all side effects need to be reported immediately to the medical team treating the patient's Fabry disease.

Kidney issues are another concern when treating patients with Fabry disease, and the Agalsidase Beta course of treatment must be conducted with a nephrologist on the comprehensive medical team that is coordinating the patient's medical care.

Storage

This drug is administered at a healthcare facility under the advisement of the patient's physician and pharmacists. The drug should not be kept in the home, nor should it be administered outside of a full-service treatment facility due to the risks inherent with infusions.

The drug is produced as a powder that is then implemented via injection and requires the expertise of a pharmacist to prepare the drug for infusion. With any type of injection, there is a risk of infection, and all care must be taken to ensure that the drug is stored in a completely sterile environment.

Summary

Fabry disease is a condition that is genetic in nature and can have an adverse effect on the kidneys and other vital organs. Agalsidase Beta is an intravenous drug that replaces the alpha-galactosidase A. The drug is administered in a healthcare facility because it is an infusion and such a delivery method can cause serious side effects. Aglasidase Beta is administered intravenously with 1mg of the drug given for every 1kg of body weight. A complementary treatment for infusion reactions must also be given during the course of treatment with Agalsidase Beta.

The side effects can range from minor skin irritations to depression and mitral valve prolapse. Because the drug is given in a healthcare facility it is important to take advantage of the advice of the medical team and follow any strategies to lessen and alleviate the side effects of the drug. The main concern is the health of the kidneys as Fabry disease can cause severe damage to the kidneys and patients may require a kidney transplant in severe cases.

Use of Aglasidase Beta has not been adequately studied for the use in children, by pregnant women, or mothers who are breastfeeding. Due to the low incidence of Fabry disease in women, it is unlikely that the use of this drug in pregnant or breastfeeding women will be conducted. In the event that the patient is a woman seeking treatment for Fabry disease, the doctor can make the determination on the appropriateness of the drug for their situation. Likewise, there are few studies in geriatric patients due to the life expectancy age of 58 in men suffering from Fabry disease.

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Last Reviewed:
December 10, 2017
Last Updated:
April 04, 2018
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