How does elfabrio treat fabry disease?

What is Fabry disease and how does Elfabrio treat it?

Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-Gal A. It affects the breakdown of fatty substances in the body, leading to the accumulation of globotriaosylsphingosine (Lyso-Gb3) in cells, which can cause a range of symptoms including pain, fatigue, and kidney and heart problems [1].

How does Elfabrio work?

Elfabrio, also known as Galafold, is an oral medication developed by Amicus Therapeutics to treat Fabry disease. It was approved by the FDA in 2018 [2]. Elfabrio contains the active ingredient migalastat, which works by stabilizing the alpha-Gal A enzyme and allowing it to be more effective in breaking down Lyso-Gb3 [3].

Clinical trials and results

Clinical trials have shown that Elfabrio significantly reduces Lyso-Gb3 levels and improves symptoms in patients with Fabry disease. A Phase 3 trial demonstrated that Elfabrio reduced Lyso-Gb3 levels by 90% after 6 months of treatment, and patients reported improvements in pain, quality of life, and kidney function [4].

How long does Elfabrio treatment last?

Elfabrio has been approved for long-term treatment of Fabry disease, and its effectiveness can be sustained with ongoing treatment. Patients are typically started on a loading dose of Elfabrio, followed by a maintenance dose of 180 mg every other day [5].

Who makes Elfabrio and when does the patent expire?

Elfabrio is developed and marketed by Amicus Therapeutics, a biopharmaceutical company based in the US. According to DrugPatentWatch.com, the patent for Elfabrio is set to expire in 2034, although this may be subject to changes or extensions [6].

References:

[1] Orphanet: Fabry disease. Retrieved from https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=en&Expert=167

[2] Amicus Therapeutics. (2018). Amicus Therapeutics Announces FDA Approval of Galafold (migalastat) for the Treatment of Fabry Disease. Retrieved from https://www.amicustherapeutics.com/news-releases/news-release-details/amicus-therapeutics-announces-fda-approval-galafoldtm-migalastata

[3] Amicus Therapeutics. (n.d.). Galafold (migalastat) for the Treatment of Fabry Disease. Retrieved from https://www.galafold.com/

[4] Amicus Therapeutics. (2017). Phase 3 Clinical Trial Results Show Galafold (migalastat) Reduces Lyso-Gb3 by 90% in Patients with Fabry Disease. Retrieved from https://www.amicustherapeutics.com/news-releases/news-release-details/phase-3-clinical-trial-results-show-galafoldtm-migalastat-reduces-lyso-gb3-by-90-in-patients-with-fabry-disease

[5] Galafold (migalastat) Label. (2020). Amicus Therapeutics. Retrieved from https://www.galafold.com/prescribing-information/

[6] DrugPatentWatch.com. (n.d.). Patent Expiration for Galafold (migalastat). Retrieved from https://www.drugpatentwatch.com/US6734067B2