FAQs About Replagal

FAQs About Replagal
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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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Fabry Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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One comment

  1. Susanne says:

    I have a variant of uncertain significance.Not uncertain to me as I suffer all Fabry symptoms. Replagal has has been effective for me.My son has been refused ERT even though he has the same mutation. I would be prepared to share my dose with him, rather than watch him suffer without help. We could have the treatment together once a month ,at no extra cost .

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