fabrazyme

How Was Its Use First Established?

• Produced by ''Sanofi Genzyme''
• founded by George Whitesides and Sheridan Snyder
• specialty care global business unit of Sanofi
• focuses on finding solutions to rare diseases, rare blood disorders, multiple sclerosis, oncology, and immunology
• goal is to make a positive impact on the lives of patients around the world and continue in their progress as much as they can
• the effectiveness of Fabrazyme was recognized and implemented all around the world to treat patients with Fabry disease

What Is The Source Of the Enzyme?

• agalsidase beta (active substance in Fabrazyme) is a copy of the human enzyme which is produced by ''recombinant DNA technology''
• made by cells that received a gene which enables them to produce the enzyme
• Fabrazymes effectiveness was tested on almost 90 different individuals of various ages who had Fabry disease and the results were a success

Before the Production Of Fabrazyme?

• Replagal, produced by ''Takeda'' was used to treat patients with Fabry disease before Fabrazyme
• release date: 03/08/2001
• medicine that contains the active substance agalsidase alfa
• made into a solution for infusion into a vein
• proved to be slightly less effective then Fabrazyme (explained in pros and cons chart)
• before Replagal, there was no cure or medicine to help patients with Fabry disease

Why Is It Important?

• as mentioned before, Fabrazyme helps by lowering the build up of GL-3 in body cells
• this fat causes signs and symptoms that affect parts of the body
• Fabry disease causes pain in hands and feet, groups of small, red spots on skin, decreases ability to sweat, cloudiness in front of eyes, issues with gastrointestinal system, hearing loss, life-threatening problems with kidney damage, heart attacks and strokes.
• these serious symptoms and life-threatening complications can be stopped with the usage of Fabrazyme

What Is It Used For?

Fabrazyme

• Fabrazyme is used for treating patients with Fabry disease
• it replaces a missing enzyme and lowers the amount of globotriaosylceramide (GL-3) which builds up in certain cells and some that line the blood vessels of the kidney (caused negative affects for patients)
• most patients received results of lower GL-3 levels after several infusions of Fbrazyme
• levels went down to normal or near normal

Agalsidase Beta;

How Has The Use Of Enzymes Increased Efficiency?

Approved on: 4/24/2003

• Fabry disease is an inherited condition caused by complications in the GLA gene (provides instructions for making an enzyme called alpha-galactosidase A).
• alpha galactosidase A breaks down GL-3
• patients with Fabry disease cannot make enough alpha-galactosidase A which is why there is a build up of GL-3 as mentioned before
• agalsidase beta is added to Fabrazyme and is a copy of the missing enzyme alpha-galactosidase A
• treatment with Fabrazyme includes infusing it into the bloodstream once every two weeks and aids in the breakdown of GL-3

By: Fatima Sultana

Reasons For Choosing This Topic

References

I searched up a couple of the topics and examples given and was interested with ''Fabrazyme'' since it was related to helping a patient with fabry disease which is a disease I had never heard of before, though it is rare. The fact that we are able to make such a cure that can help someone by replacing and growing a new enzyme is something I had known of before but did not know any examples of or how it worked. By doing this project I was able to learn a lot and know that we as humans are capable of doing great things in order to help each other if we have willingness to achieve it.

Pros

Cons

• Anonymous. (2018, November 29). Replagal. Retrieved October 18, 2020, from https://www.ema.europa.eu/en/medicines/human/EPAR/replagal
• Brian Murphy, P. (2020, October 16). Fabrazyme vs. Replagal for the Treatment of Fabry Disease. Retrieved October 18, 2020, from https://fabrydiseasenews.com/2020/10/16/fabrazyme-vs-replagal-for-the-treatment-of-fabry-disease/
• Drug Approval Package: FABRAZYME (AGALSIDASE BETA). (n.d.). Retrieved October 18, 2020, from https://www.accessdata.fda.gov/drugsatfda_docs/nda/2003/103979_toc.cfm
• Fabrazyme - Uses, Side Effects, Interactions. (n.d.). Retrieved October 18, 2020, from https://www.medbroadcast.com/drug/getdrug/fabrazyme
• Fabrazyme® (agalsidase beta). (n.d.). Retrieved October 18, 2020, from https://www.fabrazyme.com/about-fabrazyme
• Fabry disease: MedlinePlus Genetics. (2020, August 18). Retrieved October 18, 2020, from https://medlineplus.gov/genetics/condition/fabry-disease/
• Sanofi Genzyme Homepage - Sanofi Genzyme. (n.d.). Retrieved October 18, 2020, from https://www.sanofigenzyme.com/
• Wharf, C. (2013). Fabrazyme [PDF]. London: European medicines agency.

• people allergic to agalsidase beta cannot use it
• In clinical studies, 59% of patients experienced infusion-associated reactions during treatment
• People with advanced Fabry disease may have heart problems, putting them at a higher risk for severe complications from infusion-associated reactions
• symptoms of a serious allergic reaction (ex. shortness of breath; difficulty breathing; wheezing; hives; swelling of the face, lips, or throat)
• skin rash
• stomach discomfort
• symptoms of an infection (cough, runny nose, sore throat)
• toothache
• back pain
• decreased hearing
• depression
• difficulty sleeping
• pain, including muscle and neck pain
• ringing in the ears

• causes more reduction in Gb3 in the blood then Replagal
• has a greater affect on the left ventrical of the heart then Replagal
• causes a greater response from immune system then Replagal since it uses CHO instead of a human cell line
• there are two ways to address infusion related negative reactions
• 1. before the infusion
• people who have shown signs of reacting negatively to infusions are pretreated with acetaminophen before their next infusion which helps prevent future reactions
• 2. during the infusion
• slowing down the infusion rate or taking pauses during it helps relieve some symptoms
• emergency medical treatment is available for severe reactions
• can be taken at home after several infusions