agalsidase beta

Action and Spectrum

agalsidase beta is an enzyme replacement therapy used to treat Fabry disease. This rare inherited disorder affects the body’s ability to break down a fat called globotriaosylceramide (Gb3 or GL-3). In Fabry disease, the body lacks an enzyme called alpha-galactosidase A, which is necessary to break down Gb3. As a result, Gb3 accumulates in the body’s cells, leading to various symptoms and complications. 

agalsidase beta is a recombinant form of alpha-galactosidase A. It replaces the missing enzyme in the body, allowing it to break down Gb3 and reduce its accumulation in cells. By reducing the accumulation of Gb3, agalsidase beta can help improve the symptoms and complications of Fabry disease. 

agalsidase beta is specifically indicated for the treatment of Fabry disease in adults and children over the age of 6 years. It is not adequate for the treatment of other conditions or disorders. 

Drug Interaction

Adverse Reaction

Frequency defined:  

>10% 

Adults 

Chills 

Headache 

Paresthesia 

Peripheral edema 

Rash 

Myalgia 

Pain 

Hypertension 

Upper respiratory tract infection 

Pyrexia 

Cough 

Fatigue 

Dizziness 

Pain in extremity 

Lower respiratory tract infections 

Back pain 

Pediatric 

Abdominal Pain 

Fever 

Vomiting 

Diarrhea 

Dizziness 

Headache 

Pharyngitis 

Nausea 

Arthralgia 

Rhinitis 

1-10% 

Adults 

Tachycardia 

Increased blood creatinine 

Dyspnea 

Burning sensation 

Depression 

Hypoacusis 

Fungal infection 

Hot flush 

Pruritis 

Excoriation 

Tinnitus 

Fall 

Anxiety 

Wheezing 

Chest discomfort 

Viral infection 

Post-marketing reports 

Hyperhidrosis 

Arthralgia 

Increased lacrimation 

Renal failure 

Leukocytoclastic vasculitis 

Sepsis 

Pneumonia 

Asthenia 

Cardiac failure 

Black Box Warning

Contraindication / Caution

agalsidase beta is contraindicated in individuals with known hypersensitivity to the drug or its components. 

There are a few precautions to be aware of when taking agalsidase beta. It is essential to inform your healthcare provider if you have any allergies, including allergies to foods, dyes, or preservatives. 

agalsidase beta may cause allergic reactions, which can range from mild to severe. Symptoms of an allergic reaction may include rash, itching, swelling, trouble breathing, or dizziness. If you experience these symptoms, you should immediately stop taking agalsidase beta and seek medical attention. 

agalsidase beta may not be suitable for all individuals, and your healthcare provider will consider your specific situation when determining whether this medication is appropriate for you. 

Pregnancy / Lactation

Pregnancy consideration: Insufficient data available 

Lactation: Excretion of the drug in human breast milk is unknown 

Pregnancy category: 

Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester.   

Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women. 

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data available with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.    

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: There is no data available for the drug under this category 

Pharmacology

Pharmacology

agalsidase beta is an enzyme replacement therapy used to treat Fabry disease. In Fabry disease, the body lacks an enzyme called alpha-galactosidase A, which is necessary to break down Gb3. As a result, Gb3 accumulates in the body’s cells, leading to various symptoms and complications. 

An agalsidase beta is a recombinant form of alpha-galactosidase A. It replaces the missing enzyme in the body, allowing it to break down Gb3 and reduce its accumulation in cells. By reducing the accumulation of Gb3, agalsidase beta can help improve the symptoms and complications of Fabry disease. 

Pharmacodynamics 

The main pharmacodynamic effect of agalsidase beta is the reduction of Gb3 accumulation in cells, which can improve the symptoms and complications of Fabry disease. agalsidase beta has been shown to reduce the size of kidney glomeruli and improve kidney function, reduce the frequency and severity of angiokeratomas (small, red-purple bumps on the skin), and improve cardiovascular function in patients with Fabry disease. It may also improve some patients’ neurological symptoms, such as pain. 

Pharmacokinetics 

The pharmacokinetic parameters refer to the absorption, distribution, elimination, and clearance of agalsidase beta in the body. 

Absorption 

The peak plasma concentration of agalsidase beta ranges from 0.6 to 29.7 mcg/mL following a 0.3-1 mg/kg dose. 

Distribution 

The volume of distribution (Vd) of agalsidase beta at steady-state ranges from 81 to 330 mL/kg following a 0.3-1 mg/kg dose.  

Metabolism 

Agalsidase beta is metabolized in the liver after it is administered. The drug is rapidly cleared from circulation, with a half-life of approximately 2.5 hours. It is metabolized by hydrolysis, which involves breaking down the drug into smaller molecules. 

Elimination/Excretion 

The half-life of agalsidase beta ranges from 45 to 102 minutes following a 0.3-1 mg/kg dose. This indicates that the drug is rapidly cleared from the body. The clearance of agalsidase beta ranges from 0.8 to 4.6 mL/min/kg following a 0.3-1 mg/kg dose. 

Adminstartion

Administration

Agalsidase beta is administered as an intravenous (IV) injection. It is usually given once every other week at a dose of 0.2 mg/kg as a 60-minute IV infusion. The dose may be adjusted based on the patient’s needs and treatment response. 

Before administering agalsidase beta, the drug should be prepared and diluted according to the instructions provided. The reconstituted solution should be inspected for particulate matter and discoloration and discarded if these are present. The solution should be diluted with 0.9% NaCl to a total volume based on the patient’s weight. The infusion rate and duration may vary based on the patient’s weight and tolerance to the drug. 

It is essential to have appropriate medical support measures available and to administer antipyretics before the infusion. In the event of an infusion-associated reaction, the infusion rate should be slowed. 

Agalsidase beta does not contain preservatives and should be used immediately after reconstitution. Unused vials should be refrigerated and discarded after the expiration date. Reconstituted vials and diluted solutions should be used immediately or refrigerated for up to 24 hours. 

Following the recommended administration guidelines and your healthcare provider’s instructions is essential when using agalsidase beta. If you have any questions or concerns about the administration of this medication, you should speak with your healthcare provider. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: agalsidase beta 

Pronounced: [ a-GAL-sih-daze-BAY-tah ]  

Why do we use agalsidase beta? 

agalsidase beta is a medication that is used to treat Fabry disease, which is a rare inherited disorder that affects the body’s ability to break down a type of fat called globotriaosylceramide. People with Fabry disease do not produce enough of an enzyme called alpha-galactosidase A, which is necessary for the proper metabolism of globotriaosylceramide. As a result, the fat builds up in cells and tissues, leading to various symptoms, including kidney, heart, and nerve problems. agalsidase beta is a genetically engineered form of alpha-galactosidase A that replaces the missing enzyme in people with Fabry disease. It is given by intravenous infusion (through a vein) to help the body break down the excess fat and prevent further damage to cells and tissues.