What is Fabrazyme used for?
Fabrazyme is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. One goal in the treatment of Fabry disease is to clear and prevent future buildup of a fatty substance called GL-3, which can accumulate in key organs and cause organ damage.
What type of drug is Fabrazyme?
Drug details Fabrazyme is an enzyme replacement therapy. Its active ingredient is an enzyme (a type of protein) called agalsidase beta. The drug works by replacing a certain enzyme that either isn’t produced by your body, or that doesn’t work correctly.
Who manufactures vpriv?
Velaglucerase alfa, sold under the brand name Vpriv and manufactured by Shire plc, is a hydrolytic lysosomal glucocerebroside-specific enzyme, which is a recombinant form of glucocerebrosidase indicated as a long-term enzyme replacement therapy for those with Gaucher disease Type 1.
Is vpriv made from human ovaries?
Currently, three approved ERTs exist for Gaucher disease, Sanofi’s Cerezyme, which is made from Chinese hamster ovary (CHO) cells, Pfizer’s Elelyso, which is made using carrot cells, and Vpriv by Shire (now Takeda), which is produced in a human fibroblast cell line.
How is Fabry diagnosed?
Your healthcare provider may order tests to diagnose Fabry disease, including:
- Enzyme assay: This test measures alpha-GAL enzymes in blood.
- Genetic: Because females with Fabry disease can have normal levels of alpha-GAL enzymes, providers use genetic testing (DNA sequencing) to identify the GLA gene mutation.
What is another name for Fabry disease?
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases.
What drug class is Fabrazyme?
Fabrazyme belongs to a class of drugs called Enzymes, Metabolic.
What is the goal of enzyme therapy for Gaucher disease?
How Does Enzyme Replacement Therapy Work? ERT balances low levels of glucocerebrosidase (GCase) enzyme with a modified version of the enzyme. This enzyme breaks down glucocerebroside, the fatty chemical that accumulates in the body of patients with Gaucher disease.
What causes Fabry disease?
Fabry’s disease is caused by a change in the GLA gene, which helps create an enzyme (alpha-galactosidase) that breaks down globotriaosylceramide. Men are more likely to have the condition. The condition is rare and occurs in an estimated 1 out of every 40,000 to 60,000 men.
What are Fabry symptoms?
Fabry disease symptoms include:
- Numbness, tingling, burning or pain in the hands or feet.
- Extreme pain during physical activity.
- Heat or cold intolerance.
- Abnormal opacity of the eye (cornea), which does not change someone’s vision.
- Dizziness.
- Flu-like symptoms, including fatigue, fever and body aches.
What causes Fabry?
Fabry disease is caused by variants (also known as mutations) in the GLA gene. This gene provides instructions for making an enzyme called alpha-galactosidase A. This enzyme is active in lysosomes , which are structures that serve as recycling centers within cells.
When to use Replagal for Fabry disease?
4. Clinical particulars Replagal is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease (α-galactosidase A deficiency). Replagal treatment should be supervised by a physician experienced in the management of patients with Fabry Disease or other inherited metabolic diseases.
What is Replagal?
Replagal is medicine that contains the active substance agalsidase alfa. It is available as a concentrate to be made into solution for infusion (drip) into a vein. What is Replagal used for? Replagal is used to treat patients who have Fabry disease, a rare inherited disorder.
Who should take Replagal?
Only a doctor who has experience in treating patients with Fabry disease or other inherited metabolic diseases should give Replagal. Replagal is given once every 2 weeks as an infusion of 0.2 mg per kilogram body weight over 40 minutes. It is intended for long-term use. How does Replagal work? Replagal is an enzyme-replacement therapy.
What happens if I stop using Replagal?
If you stop using REPLAGAL, the level of the enzyme which is responsible for Fabry Disease will remain low and the symptoms of the disease will not be treated.