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US 12109205

Methods of treating Fabry patients having renal impairment

Current assignee: Amicus Therapeutics Inc

Added 9/21/2026, 9:43:02 AM

At a glanceNo PTAB challengesNo litigation on file

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Patent summary

Title, assignee, inventors, filing/issue dates, abstract, and a plain-language overview of the claims.

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Patent Overview: US 12109205 B2

Title: Methods of treating Fabry patients having renal impairment

Patent number: US 12109205 B2
Application number: US 17/078,840
Inventors: Jeff Castelli; Elfrida Benjamin
Assignee: Amicus Therapeutics Inc. (original and current assignee, per the source)
Priority date: 2017-05-30
Filing date: 2020-10-23
Publication date (grant): 2024-10-08
Legal status: Active, with an adjusted expiration of 2039-05-02 (source notes legal status is an assumption, not a legal conclusion).

Purpose: The patent relates generally to using pharmacological chaperones to treat Fabry disease, particularly in patients with varying degrees of renal impairment. Fabry disease is described as a lysosomal storage disorder caused by a mutation in the GLA gene encoding α-galactosidase A (α-Gal A), leading to accumulation of the substrate globotriaosylceramide (GL-3). The described treatment uses migalastat, a small-molecule chaperone, and can include stabilizing renal function, reducing left ventricular mass index (LVMi), reducing plasma lyso-Gb3, and/or increasing α-Gal A activity.

Plain-language scope: One described aspect is a method of treating Fabry disease in a patient with renal impairment by administering an effective amount of migalastat or a salt thereof once every other day. The effective amount is described as about 100 mg to about 150 mg free base equivalent (FBE); specific examples include about 123 mg FBE, about 123 mg migalastat free base, and about 150 mg migalastat hydrochloride. The patient may have mild, moderate, or severe renal impairment; may be ERT-experienced or ERT-naïve; and may have proteinuria below 100 mg/24 hr, 100–1,000 mg/24 hr, or above 1,000 mg/24 hr. Administration may be oral (tablet, capsule, or solution) for at least 28 days, 6 months, or 12 months. Another aspect covers use of migalastat to stabilize renal function in a Fabry patient with renal impairment.

Source: https://patents.google.com/patent/US12109205/en

What the excerpt does not establish: It does not provide the full claims, complete specification, examples, or data; the excerpt is expressly partial and directs readers to the source for omitted text. It also does not establish claim scope, validity, or the accuracy of dates, assignee listings, or legal status, which the source itself identifies as assumptions or non-conclusions.

Generated 10/1/2026, 10:01:54 PM

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Assignment history

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Prior art

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