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What is Fabry disease?

Fabry disease, also known as Anderson-Fabry disease, is a rare genetic disorder that affects many organs and systems in the body. It is caused by a deficiency of an enzyme called alpha-galactosidase A (AGA), which leads to a buildup of a fatty substance called globotriaosylceramide (Gb3) in various cells and tissues. The buildup of Gb3 can cause damage and problems in the affected organs, leading to a variety of symptoms and complications.


What are the symptoms and diagnosis of Fabry disease?

Fabry disease is inherited in an X-linked pattern, meaning that it primarily affects males. Females who inherit the mutated gene can also be affected but often have milder symptoms. The severity of symptoms can vary widely between individuals, even within the same family.


Symptoms can begin in childhood or adolescence and may include:

  • Pain and burning sensations in the hands and feet

  • Skin rashes

  • Corneal opacities (scarring of the clear front layer of the eye)

  • Gastrointestinal symptoms such as abdominal pain or diarrhea

  • Hearing loss

  • Kidney problems

  • Cardiac abnormalities such as irregular heartbeats, heart attacks, and heart failure

The diagnosis of Fabry disease is often delayed because many of the symptoms are nonspecific and can mimic other conditions. However, it is important to diagnose and treat the disease early to prevent or delay the onset of complications.


The diagnosis is made by measuring the activity of AGA in the blood or through genetic testing to identify mutations in the GLA gene, which provides instructions for making the AGA enzyme.


What drugs are used to treat Fabry disease?

Treatment of Fabry disease typically involves enzyme replacement therapy (ERT), where a synthetic version of the missing enzyme is given through intravenous infusion. ERT can help reduce the accumulation of Gb3 and improve symptoms and quality of life. Other treatments may include medications to manage pain, blood pressure, and other symptoms, as well as lifestyle modifications such as a low-fat diet and regular exercise.


There are currently three drugs on the market for Fabry Disease: Fabrazyme (agalsidase beta) from Sanofi, Galafold (migalastat hydrochloride) from Amicus Therapeutics, and the recently approved Elfabrio (pegunigalsidase alfa-iwxj) from Chiesi and Protalix.

Drug Name

Fabrazyme


Galafold


Elfabrio


Manufacturer

Sanofi

Amicus Therapeutics

Chiesi/Protalix

Active Ingredient

Agalsidase beta

Migalastat hydrochloride

Pegunigalsidase alfa-iwxj

Inactive Ingredients

Mannitol, sodium phosphate monobasic, monohydrate, and sodium phosphate, dibasic, heptahydrate

Shellac, magnesium stearate, starch, corn, gelatin, FD&C Blue No. 2, titanium dioxide, ferrosoferric oxide, potassium hydroxide

Anhydrous citric acid, sodium chloride, sodium citrate, and water for injection

Indication

Treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease

Treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data

Treatment of adults with confirmed Fabry

disease


Side Effects


  • Upper respiratory tract infections

  • Chills

  • Pyrexia

  • Headache

  • Cough

  • Paresthesia

  • Fatigue

  • Peripheral edema

  • Dizziness

  • Rash



  • Headache

  • Nasopharyngitis

  • Urinary tract infection

  • Nausea

  • Pyrexia

  • Abdominal pain

  • Back pain

  • Cough

  • Diarrhea

  • Epistaxis



  • Infusion-associated reactions

  • Nasopharyngitis

  • Headache

  • Diarrhea

  • Fatigue

  • Nausea

  • Back pain

  • Pain in extremity

  • Sinusitis


Animal-Free?

NO (Chinese Hamster Ovary Cells)

NO (Shellac and Gelatin)

YES

*For full prescribing information, visit https://www.fabrazyme.com/, https://www.galafold.com/, and https://elfabrio.com/


Is there an animal-free treatment option for Fabry disease?

We are thrilled to announce that Elfabrio from Chiesi and Protalix is verified animal-free! Unlike its competitors, the manufacturing process and final product of Elfabrio did not involve any animal-derived ingredients. The active ingredient in Elfabrio, pegunigalsidase alfa, is developed by Protalix using plant cell culture and is a chemically modified version of the recombinant alpha-galactosidase-A protein.


Conclusion

Fabry disease is a rare genetic disorder that can cause a range of symptoms and complications. However, early diagnosis and treatment can help manage the disease and improve patients’ quality of life. It’s also important to note that advances in technology and research have led to the development of new treatments like Elfabrio, which is an animal-free option for patients.


We are excited to see more animal-free treatments like Elfabrio being developed, and we will continue to support and promote companies that prioritize animal welfare and environmental sustainability in their research and development efforts!

 

Disclaimer: The product and/or information provided on VeganMed is of a general nature and is not intended to be a substitute for professional medical advice, diagnosis, or treatment. We do not lab test the products to confirm that they are free from animal ingredients, and it is possible that the formulation and ingredients could have changed. Always seek the advice of your physician or other qualified healthcare provider with any questions you may have regarding a medical condition or product. The information provided in this post is accurate and up to date as of the date it was written. However, please note that circumstances and facts may change over time, and new information may become available that could alter the accuracy or relevance of the content. We encourage readers to verify and cross-reference any information provided here with trusted sources or consult relevant professionals for the most current and accurate updates.

 

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