Agalsidase beta is a medication used to treat a rare genetic disorder. It provides a protein missing or deficient in individuals with this inherited condition. It addresses the disorder’s underlying issue by supporting cellular processes.
The Condition Agalsidase Beta Treats
Agalsidase beta is prescribed for Fabry disease, a rare genetic disorder. It stems from a mutation in the GLA gene. This mutation leads to a deficiency of the alpha-galactosidase A enzyme.
This enzyme deficiency causes the accumulation of a fatty substance, globotriaosylceramide (Gb3 or GL-3), within cells and organs. This build-up can damage organ systems throughout the body. Symptoms may affect kidneys, heart, brain, skin, and the nervous system, illustrating the widespread impact of the disorder.
How Agalsidase Beta Functions
Agalsidase beta is an enzyme replacement therapy (ERT). It is a manufactured version of the human alpha-galactosidase A enzyme, produced through recombinant DNA technology. This engineered enzyme mimics the natural human enzyme deficient in Fabry disease patients.
When administered, agalsidase beta replaces the missing enzyme in affected individuals. This allows the body to break down and clear the accumulated GL-3 from cells and tissues. By removing this fatty substance, agalsidase beta helps prevent or reduce the progressive damage to various organs caused by its build-up, thereby addressing the root cause of Fabry disease.
Receiving Agalsidase Beta Treatment
Agalsidase beta is administered through intravenous (IV) infusion. Infusions are typically given once every two weeks. Each infusion is generally at least 90 minutes, though it can be adjusted based on individual patient tolerability and weight.
Patients may experience infusion-associated reactions (IARs) during or after the treatment. Reactions can include fever, chills, rash, headache, dizziness, nausea, vomiting, or muscle pain. Healthcare providers may administer pre-treatment medications, such as antihistamines or antipyretics, to manage or reduce these reactions.
Severe allergic reactions, including anaphylaxis, are possible with agalsidase beta treatment. Infusions are conducted in a healthcare setting where medical support measures, including cardiopulmonary resuscitation equipment, are readily available. Patients undergo ongoing monitoring, which may include assessing kidney and heart function, as well as tracking their immune response to the medication. This treatment is long-term, aiming to manage the progression of Fabry disease and mitigate its effects over time.