Amicus Therapeutics has been granted a patent for methods of treating Fabry disease by enhancing a-galactosidase A levels in patients with mutations in the gene encoding the enzyme. The patent covers administering pharmacological chaperones to patients with Fabry disease, offering new treatment options. GlobalData’s report on Amicus Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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According to GlobalData’s company profile on Amicus Therapeutics, Human telomerase RT biomarker was a key innovation area identified from patents. Amicus Therapeutics's grant share as of January 2024 was 40%. Grant share is based on the ratio of number of grants to total number of patents.

Treating fabry disease using pharmacological chaperones for a-galactosidase a

Source: United States Patent and Trademark Office (USPTO). Credit: Amicus Therapeutics Inc

A recently granted patent (Publication Number: US11833164B2) discloses a method for treating Fabry disease in a subject by accessing mutation information corresponding to the subject, identifying specific mutations in the a-galactosidase A gene. The method involves querying a data store to determine the presence of mutations from a predefined group and administering a therapeutically effective dose of migalastat or its salt to patients with specific mutations, such as Y184S, N228H, or T412I, every other day. The administration of migalastat aims to enhance a-galactosidase A activity, reduce plasma lyso-Gb3 levels, decrease GL-3 accumulation, alleviate left ventricular hypertrophy, and improve renal function in patients with Fabry disease.

The patent also outlines the method of administering migalastat or its salt at a specific dosage, such as about 123 mg free base equivalent every other day, to patients with the identified mutations. Additionally, the patent specifies the potential benefits of the treatment, including the reduction of plasma lyso-Gb3, GL-3 accumulation, left ventricular hypertrophy, and improvement in renal function. The method aims to address the underlying genetic mutations associated with Fabry disease and provide targeted treatment to patients with specific mutations, highlighting the potential for personalized medicine in managing this rare genetic disorder effectively.

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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies