September 30, 2022

Fabrayzne: Overview, Side Effects & More

Specialty Infusion Blog Elan Katz

Fabrazyme is a prescription drug, and its generic is Agalsidase beta. To treat Fabry disease, it got FDA approval to be used in adults and children 2 years of age and older.

An uncommon hereditary illness called Fabry disease (passed down genetically through families). This condition is characterized by fat accumulation in the body brought on by a gene anomaly. This may cause symptoms that range from minor to severe and even fatal.

Indications for Fabrazyme

Patients with Fabry disease, a rare genetic condition, are treated with Fabrazyme. Many symptoms may be present in Fabry disease patients, including severe disorders, renal failure, cardiac issues, and stroke. The alpha-galactosidase is an enzyme that is insufficient in people with Fabry disease. Usually, this enzyme degrades a fatty molecule known as globotriaosylceramide (GL-3). Without this enzyme, GL-3 cannot be degraded and accumulates in the body’s cells, including kidney cells.

Because there are few people with Fabry disease, it is regarded as a “rare illness,” and Fabrazyme was named an “orphan drug” (a medication used in uncommon disorders) on August 8, 2000. Only those with a valid prescription may purchase the medication.

Approval of Fabrazyme

Fabrazyme therapy may provide long-term therapeutic advantages for people with Fabry disease. The Committee for Medicinal Products for Human Use (CHMP) was the decision. The CHMP determined that for long-term enzyme replacement treatment that in individuals with a confirmed diagnosis of Fabry disease, the advantages of Fabrazyme outweigh the risks. Fabrazyme was, therefore, recommended for commercial authorization by the board.

Fabrazyme was first approved under “exceptional circumstances” since there wasn’t enough information available at the time of approval due to the condition’s rarity.

Fabrazyme Mechanism of Action

Using Fabrazyme is a part of enzyme replacement treatment. Patients who need an enzyme are given it via enzyme replacement therapy. Alpha-galactosidase A, a human enzyme absent in those with Fabry disease, is replaced with Fabrazyme. Helped by “recombinant DNA technology,” the active ingredient in Fabrazyme, agalsidase beta, is created by a cell that has been given a gene (DNA) that enables it to generate the enzyme. The substitute enzyme aids in the breakdown of GL-3 and prevents it from assembling (accumulating) in the patient’s cells.

Agalsidase beta, an enzyme (a protein subclass), is an active component. This component works by substituting an enzyme either not created by your body or does not function properly.

Powdered Fabrazyme can be combined with a liquid to form a solution. A medical expert will administer the medication via intravenous (IV) infusion. There are two strengths of Fabrazyme: 5 mg and 35 mg.

The usual dose of Fabrazyme injection is every two weeks. The first dose is usually given as an infusion in a clinic or your doctor’s office. In the future, you may be able to have infusions from a doctor who visits your house.

Side effects of Fabrazyme

Severe Allergic and Hypersensitivity Reactions

During the infusion of Fabrazyme after its approval or the clinical studies, anaphylactic (allergic) or severe hypersensitive responses were seen in around 1% of patients. Patients have had potentially fatal extreme anaphylactic (allergic) or severe hypersensitive reactions when receiving Fabrazyme infusions.

The following are the allergic reactions that patients may experience while having an infusion of Fabrayzme.

  • The allergic reactions involved flushing, chest pain, irritation, nasal congestion, face, mouth, and neck swelling, constriction of the breathing airways, low blood pressure, hives, difficulty swallowing, rash, and breathing problems.
  • Individuals suffering from such allergic reactions need medical attention, such as heart and lung resuscitation, oxygen, intravenous fluids, hospitalization, and treatment with beta-adrenergic agonists, which help open the airways for breathing, antihistamines, and epinephrine (also called adrenaline), which aid in the reduction the body’s allergic reaction by reducing inflammation.
  • In such cases, doctors will immediately stop the Fabrazyme infusion and provide essential emergency medical assistance if you have a severe allergic or anaphylactic response. During your Fabrazyme infusion, the required medical care should be present due to the potential for serious hypersensitivity responses.

Formation of IgE antibodies due to Allergic reactions

In medical examinations, an allergic skin test response to Fabrazyme leads to developing IgE antibodies. The body’s immune system has been shown sometimes to generate IgE antibodies during an allergic response.

Adult patients with high antibody titers (units used to measure how much anti-drug-antibody your immune system produces) and those with anti-Fabrazyme antibodies were more likely to experience hypersensitivity reactions than adult patients with low antibody titers.

The doctor may want to test for IgE antibodies in case of allergic symptoms. The administration of Fabrazyme to patients who have had severe allergic reactions to Fabrazyme should only occur after considering the relevant hazards and benefits of continuing the treatment, only when under the close supervision of a licensed healthcare provider, and only when the patient has access to the necessary medical assistance.

Infusion-Associated Reactions

The patients who have had adverse responses because of infusion of Fabrazyme are given anti-fever and antihistamine drugs before their subsequent infusions. In the case of using such drugs before their infusions, some individuals have experienced infusion-related problems.

If an infusion-related response develops, the symptoms may be alleviated by decreasing the infusion rate, pausing the infusion briefly, administering extra anti-fever, antihistamine, and steroid medicine, or any combination of these.

In such cases, a healthcare provider should usually discontinue the Fabrazyme infusion straight soon and should treat symptoms if you have severe infusion-related symptoms. The standard treatment for severe responses is to provide antihistamines, corticosteroids, fluids, and oxygen intravenously. Medical care should be accessible during your Fabrazyme infusion since serious infusion-associated events may occur.

Other Common Side Effects

Other common side effects due to Fabrazyme include burning or tingling feeling, upper respiratory tract infection, chills, fever, headache, cough, weariness, swelling in the legs, disorientation, and rashes recorded in 20% or more of patients.

Follow Us On Instagram