United States Patent 11,202,767: A Comprehensive Analysis
Overview of the Patent
United States Patent 11,202,767, titled "Methods of treating urea cycle disorders and maple syrup urine disease," was issued on December 21, 2021. This patent is assigned to Acer Therapeutics Inc. and involves innovative methods for treating specific metabolic disorders using a particular pharmaceutical composition.
Inventors and Assignees
The patent was invented by Leah E. Appel, Joshua R. Shockey, and D. Christopher Schelling, and is assigned to Acer Therapeutics Inc., a company specializing in the development of treatments for rare and life-threatening diseases[2][4].
Patent Expiration Dates
The patent is set to expire on October 17, 2036, which is approximately 20 years from the date of filing, aligning with standard patent duration[2].
Scope of the Patent
Diseases Treated
This patent focuses on methods for treating urea cycle disorders and Maple Syrup Urine Disease (MSUD), both of which are inborn errors of metabolism. Urea cycle disorders involve defects in the enzymes that remove ammonia from the blood, while MSUD is characterized by the inability to break down certain amino acids, leading to a buildup of toxic substances in the body[1][2].
Pharmaceutical Composition
The patent describes the use of sodium phenylbutyrate, a key component in the treatment of these metabolic disorders. Sodium phenylbutyrate helps in the removal of ammonia by converting it into phenylacetylglutamine, which is then excreted by the kidneys. The patent also includes formulations that make the drug more palatable, which is crucial for patient compliance, especially in pediatric cases[1][2].
Claims of the Patent
Method of Treatment
The patent claims cover specific methods of using ACER-001, a multi-particulate dosage formulation of sodium phenylbutyrate, for oral administration. These methods are designed to effectively manage urea cycle disorders and MSUD by providing a stable and consistent release of the active ingredient[4].
Formulation and Dosage
The claims include detailed descriptions of the pharmaceutical composition, including the formulation process, the use of excipients, and the manufacturing process. This ensures that the drug is not only effective but also safe and easy to administer. The multi-particulate dosage form is particularly noted for its ability to improve patient compliance due to its palatability[1][2].
Additional Uses
While the primary focus is on urea cycle disorders and MSUD, the patent also hints at potential applications in treating other conditions, such as neurodegenerative disorders like Parkinson's disease, spinal muscular atrophy, dystonia, or inclusion-body myositis. However, these are not the primary claims of this specific patent[2].
Patent Landscape
Related Patents
Acer Therapeutics Inc. has been granted several related patents, including US Patent 11,154,521 and US Patent 11,433,041, which also deal with palatable compositions including sodium phenylbutyrate for treating various metabolic and neurodegenerative disorders. These patents collectively strengthen Acer Therapeutics' intellectual property position in this therapeutic area[2].
Generic Availability
As of the current date, there is no generic version of Olpruva (a related drug) or ACER-001 available in the United States. This lack of generic competition is due to the exclusive rights granted by the patent, which will remain in effect until its expiration in 2036[2].
Economic and Regulatory Implications
Drug Exclusivity
The patent and any associated exclusivity periods granted by the FDA ensure that Acer Therapeutics has sole marketing rights for ACER-001. This exclusivity can run concurrently with the patent term and is designed to protect the proprietary interests of the drug developer while ensuring that the drug meets stringent safety and efficacy standards[2].
Impact on Patients and Healthcare
The exclusive rights granted by this patent mean that patients with urea cycle disorders and MSUD will have access to a specific, potentially life-saving treatment, albeit at a cost that may be higher than generic alternatives. This highlights the balance between innovation and affordability in the pharmaceutical industry.
Key Takeaways
- Patent Scope: Covers methods for treating urea cycle disorders and MSUD using sodium phenylbutyrate.
- Formulation: Multi-particulate dosage form for improved palatability and patient compliance.
- Expiration: October 17, 2036.
- Related Patents: Other patents by Acer Therapeutics Inc. on similar therapeutic areas.
- Generic Availability: No generic version available until patent expiration.
- Economic Implications: Exclusive marketing rights for Acer Therapeutics Inc.
FAQs
What is the primary focus of US Patent 11,202,767?
The primary focus is on methods for treating urea cycle disorders and Maple Syrup Urine Disease using sodium phenylbutyrate.
Who are the inventors of this patent?
The inventors are Leah E. Appel, Joshua R. Shockey, and D. Christopher Schelling.
What is the significance of the multi-particulate dosage form?
The multi-particulate dosage form improves the palatability of the drug, enhancing patient compliance, especially in pediatric cases.
Are there any related patents by Acer Therapeutics Inc.?
Yes, there are related patents, including US Patent 11,154,521 and US Patent 11,433,041, which also deal with palatable compositions including sodium phenylbutyrate.
Is a generic version of ACER-001 available?
No, there is currently no generic version of ACER-001 available in the United States.
Cited Sources
- Google Patents: Methods of treating urea cycle disorders and maple syrup urine disease.
- Drugs.com: Generic Olpruva Availability.
- USPTO: Patent Claims Research Dataset.
- Biospace: Relief Announces That Collaboration Partner, Acer Therapeutics, Was Issued U.S. Patent 11,202,767.
