Is Elfabrio for Fabry Disease?

What is Elfabrio?

Elfabrio is a treatment for Fabry disease, a rare genetic disorder caused by the buildup of a type of fat called globotriaosylsphingosine (Lyso-Gb3) in the body's cells [1].

How does Elfabrio work?

Elfabrio (galafuginfor treatment of Fabry disease) works by increasing the activity of an enzyme called alpha-Gal A, which breaks down Lyso-Gb3 [1]. This helps to reduce the levels of Lyso-Gb3 in the body and alleviate symptoms of Fabry disease.

Is Elfabrio a prescription treatment?

Elfabrio was approved by the FDA in 2021 as an oral prescription treatment for adult patients with Fabry disease.

Comparison with other treatments

Elfabrio is the first oral enzyme replacement therapy (ERT) for Fabry disease. It is not a replacement for existing treatments, such as Fabrazyme and Replagal, but rather an alternative for those who require an oral form of treatment.

When is Elfabrio typically prescribed?

Elfabrio is typically prescribed for patients with a genetic deficiency of the enzyme alpha-Gal A (Fabry disease) [1].

Patent information

The patent for Elfabrio is maintained by BioMarin Pharmaceutical Inc, with various patent claims extending until as far as 2036 [2].

Costs and availability

Elfabrio is available through pharmacies in the United States and other countries following a prescription. The costs may vary depending on insurance coverage, location, and additional factors [3].

References:

[1] BioMarin Pharmaceutical Inc. (2021). Galafugin for Treatment of Fabry Disease.

[2] BioMarin Pharmaceutical Inc. (Patent No. US10744441B2). Enzyme Replacement Therapy for Fabry Disease.

[3] Drugs.com. (2022). Elfabrio Prices, Coupons & Savings Tips.