Amicus Therapeutics has been granted a patent for methods to treat Fabry disease in patients with specific mutations in a-galactosidase A. The treatment involves administering migalastat every other day, with a specified dosage. Additionally, a claim includes a molecule comprising migalastat bound to the mutated protein. 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 February 2024 was 41%. Grant share is based on the ratio of number of grants to total number of patents.
Treatment of fabry disease with migalastat for specific mutations
A recently granted patent (Publication Number: US11903938B2) discloses a molecule comprising migalastat bound to an a-galactosidase A protein with specific HEK assay amenable mutations. These mutations include a variety of amino acid substitutions such as A13T, W24R, N34T, M42K, L54F, and others. The molecule aims to enhance the stability of the a-galactosidase A protein compared to its naturally occurring form with the same mutation. Additionally, the patent covers an a-galactosidase A protein with the same mutations bound to migalastat, offering increased stability and potential therapeutic benefits for conditions like Fabry disease.
Furthermore, the patent includes a method for treating Fabry disease by administering migalastat to patients with specific a-galactosidase A protein mutations. The mutations listed in the claims, such as E87D, L89F, Y123C, and others, are targeted for their potential therapeutic effects in reducing globotriaosylceramide accumulation, decreasing plasma globotriaosylsphingosine levels, increasing white blood cell a-galactosidase A activity, reducing left ventricular mass index, and stabilizing renal function. The method involves oral administration of migalastat or its salt every other day, with dosages ranging from 123 to 300 mg, or specifically 150 mg of migalastat hydrochloride. This treatment approach is designed to address Fabry disease symptoms and improve patient outcomes, particularly in individuals with renal impairment or those who have not responded to enzyme replacement therapy.
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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
