FAQs About Replagal
Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although in use in many countries, including those of the European Union, the U.S. Food and Drug Administration (FDA) has not approved Replagal.
Following are answers to some frequently asked questions about the therapy.
How does Replagal work?
Replagal is an enzyme replacement therapy. People with Fabry disease have a mutation in the GLA gene. This means that their cells cannot produce enough functional alpha-galactosidase A enzyme. Consequently, a molecule called globotriaosylceramide (Gb3 or GL-3) builds in the body. Fabrazyme contains the enzyme agalsidase beta, which is similar to alpha-galactosidase A, and works to lower GL-3 levels in the body, which should improve symptoms.
How is Replagal given?
Replagal is administered via intravenous (directly into the bloodstream) infusion every two weeks under the supervision of a physician trained in Fabry disease. An infusion takes about 40 minutes.
How did Replagal fare in clinical trials?
A Phase 3 clinical trial (NCT00864851) assessed the safety and effectiveness of multiple dosing regimens of Replagal on heart function in adult patients. Results showed that all dosages could shrink the size of enlarged heart muscles and ease associated symptoms, including pain.
A Phase 2 clinical study (NCT00084084) tested Replagal in 17 children with Fabry disease, ages 7 to 17. The patients received 0.2 mg/kg of Replagal every week for a year, with no serious adverse side effects.
Researchers also tested Replagal manufactured in a different way in adult male patients (HGT-REP-082) and children with the disease (NCT01363492). They found no apparent differences in effectiveness or safety.
What is the recommended dose?
The recommended dose is 0.2 mg for every kilogram the patient weighs. For children and adolescents ages 7–18, a dose of 0.2 mg/kg every other week may be used.
What about side effects?
You must not take this therapy if you’re allergic to agalsidase alfa, the active substance in Replagal. In addition, side effects during or after infusion could include high fever, chills, sweating, fast heart rate, vomiting, light-headedness, hives, or swelling that could cause swallowing or breathing problems.
Most side effects are mild to moderate, although some may require treatment. Infusion-related reactions involving the heart — including heart muscle ischemia and heart failure — may occur in some patients.
Your physician may stop the infusion for some minutes until symptoms subside, and may treat them with other medicines, including antihistamines or corticosteroids. Most times, you may still receive the therapy even if the side effects occur.
If you have advanced renal disease, are pregnant or breast-feeding, speak with your physician before using Replagal.
Side effects reported in children were generally similar to those reported in adults. However, pain and infusion-related reactions including fever, difficulty breathing, and chest pain occurred more frequently in children.
Why is Replagal not approved in the U.S.?
Shire withdrew its U.S. marketing application for Replagal in 2012 after it became clear the FDA would require additional controlled clinical studies. The treatment is in use in many countries, including Canada, Russia, the U.K., Mexico, and Israel, and approved across the European Union.
Last updated: March 5, 2021
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