Detailed Analysis of the Scope and Claims of United States Patent 10,076,514
Introduction
United States Patent 10,076,514, issued on September 18, 2018, is a significant patent in the field of treating Fabry disease, a lysosomal storage disorder. This patent is assigned to Amicus Therapeutics, Inc. and covers crucial methods for the treatment and diagnosis of Fabry disease using pharmacological chaperones.
Background of Fabry Disease
Fabry disease is a genetic disorder caused by mutations in the GLA gene, which encodes the enzyme α-galactosidase A (α-Gal A). This enzyme deficiency leads to the accumulation of globotriaosylceramide (Gb3) in various tissues, resulting in severe health complications, including renal failure, cardiac issues, and neurological symptoms[4].
Patent Overview
The patent 10,076,514 focuses on methods to determine whether a patient with a lysosomal storage disorder, specifically Fabry disease, will benefit from treatment with a specific pharmacological chaperone. Here are the key aspects of the patent:
Methods for Determining Responsiveness
The patent describes in vitro methods for determining α-Gal A responsiveness to pharmacological chaperones such as 1-deoxygalactonojirimycin (DGJ) in cell lines expressing mutant forms of α-Gal A. This involves assessing the stability and activity of the enzyme in the presence of the chaperone[1].
Diagnosis of Fabry Disease
The patent also provides methods for diagnosing Fabry disease in patients suspected of having the condition. This includes genetic testing to identify mutations in the GLA gene and biochemical assays to measure α-Gal A enzyme activity[1].
Pharmacological Chaperones
Pharmacological chaperones are small molecules that help stabilize and correctly fold proteins, in this case, the mutant α-Gal A enzyme. The patent highlights the use of DGJ as a pharmacological chaperone to enhance the activity of α-Gal A, thereby alleviating the symptoms of Fabry disease[4].
Dosing Regimens
While the patent 10,076,514 itself does not detail specific dosing regimens, related patents (e.g., 10,849,889 and 11,033,538) issued to Amicus Therapeutics provide dosing regimens for administering pharmacological chaperones to treat lysosomal storage disorders. These regimens are crucial for optimizing the therapeutic efficacy and safety of the treatment[1].
Claims and Scope
The patent includes several claims that cover:
- In vitro methods for determining α-Gal A responsiveness to pharmacological chaperones.
- Methods for diagnosing Fabry disease in patients suspected of having the condition.
- Use of pharmacological chaperones such as DGJ for treating Fabry disease.
- Specific mutations in the GLA gene that are amenable to treatment with pharmacological chaperones.
These claims are designed to protect the intellectual property related to the use of pharmacological chaperones in the treatment of Fabry disease, ensuring that Amicus Therapeutics maintains exclusivity over these methods until the patent expires[1].
Patent Expiration Dates
The patent 10,076,514 is set to expire on February 12, 2029. This expiration date is critical for generic manufacturers and other pharmaceutical companies that may be interested in developing similar treatments once the patent protection lapses[1].
Litigation and Generic Challenges
The patent is part of a broader portfolio of patents held by Amicus Therapeutics that are subject to litigation and generic challenges. For instance, Aurobindo has filed an Abbreviated New Drug Application (ANDA) seeking FDA approval for a generic version of Galafold (migalastat), which is protected by several patents including 10,076,514. Amicus Therapeutics has taken legal action to protect its intellectual property rights[2].
Patent Landscape
The patent landscape surrounding 10,076,514 is complex, with multiple related patents covering various aspects of treating Fabry disease with pharmacological chaperones. Amicus Therapeutics holds a significant number of patents in this area, including those related to dosing regimens, diagnosis methods, and specific pharmacological chaperones. This extensive patent portfolio helps Amicus Therapeutics maintain a strong position in the market for Fabry disease treatments[1][2].
Patent Analytics and Claim Coverage
To manage and analyze the extensive patent portfolio, companies like Amicus Therapeutics can use patent analytics tools. These tools help in categorizing patents by claims and scope concepts, generating interactive claim charts, and identifying gaps or opportunities in the patent coverage. This is particularly useful for tracking which patents and claims are actively protecting the intellectual property and where future design opportunities or gaps exist[3].
Key Takeaways
- Treatment Methods: The patent covers in vitro methods for determining α-Gal A responsiveness to pharmacological chaperones and methods for diagnosing Fabry disease.
- Pharmacological Chaperones: DGJ is a key pharmacological chaperone used to enhance α-Gal A activity.
- Patent Expiration: The patent expires on February 12, 2029.
- Litigation: The patent is part of ongoing litigation related to generic challenges.
- Patent Landscape: The patent is part of a broader portfolio covering various aspects of Fabry disease treatment.
FAQs
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What is the primary focus of United States Patent 10,076,514?
- The primary focus is on methods to determine whether a patient with Fabry disease will benefit from treatment with a specific pharmacological chaperone and methods for diagnosing Fabry disease.
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What is the role of 1-deoxygalactonojirimycin (DGJ) in the patent?
- DGJ is used as a pharmacological chaperone to stabilize and enhance the activity of the mutant α-Gal A enzyme.
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When does the patent 10,076,514 expire?
- The patent expires on February 12, 2029.
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Why is this patent significant in the treatment of Fabry disease?
- It provides critical methods for determining patient responsiveness to pharmacological chaperones and diagnosing Fabry disease, which are essential for effective treatment.
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What is the current legal status regarding generic challenges to this patent?
- There are ongoing legal challenges, including an Abbreviated New Drug Application (ANDA) filed by Aurobindo, which Amicus Therapeutics is contesting to protect its intellectual property rights.
Cited Sources
- Generic Galafold Availability - Drugs.com
- in the united states district court - RPX Insight
- Patent Analytics | Intellectual Property Law - SLWIP
- Methods of treating Fabry disease in patients having a mutation in ... - Justia Patents
