Details for Patent: 8,354,427

Which drugs does patent 8,354,427 protect, and when does it expire?

Patent 8,354,427 protects KALYDECO, TRIKAFTA (COPACKAGED), and SYMDEKO (COPACKAGED), and is included in five NDAs.

This patent has one hundred and eighteen patent family members in twenty-six countries.

Summary for Patent: 8,354,427

Title:	Modulators of ATP-binding cassette transporters
Abstract:	The present invention relates to modulators of ATP-Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.
Inventor(s):	Van Goor; Fredrick (San Diego, CA)
Assignee:	Vertex Pharmaceutical Incorporated (Cambridge, MA)
Application Number:	12/635,927
Patent Claim Types: see list of patent claims	Use;

United States Patent 8,354,427: A Comprehensive Analysis

Overview of the Patent

The United States Patent 8,354,427, titled "Modulators of ATP-Binding Cassette Transporters," was granted to Vertex Pharmaceuticals Incorporated on January 15, 2013. This patent is a significant development in the field of pharmaceuticals, particularly in the treatment of diseases mediated by ATP-Binding Cassette (ABC) transporters, including the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)[1][4][5].

Technical Field of the Invention

The patent pertains to modulators of ABC transporters, which are a family of membrane transporter proteins. These proteins regulate the transport of various pharmacological agents, potentially toxic drugs, xenobiotics, and anions. The invention specifically focuses on modulators of CFTR, a type of ABC transporter, and methods for treating diseases associated with these transporters[1].

Background of the Invention

ABC transporters are crucial for cellular functions, using ATP to facilitate the transport of substances across cell membranes. Mutations in these transporters can lead to severe diseases, such as cystic fibrosis, which is caused by mutations in the CFTR gene. The invention aims to address these mutations by developing modulators that can restore or enhance the function of these transporters[1].

Claims of the Patent

The patent includes several claims that outline the scope of the invention:

• Modulators of ABC Transporters: The patent claims modulators of ABC transporters, including CFTR, and compositions containing these modulators.
• Methods of Treatment: It claims methods for treating diseases mediated by ABC transporters using these modulators.
• Specific Mutations: The patent specifically mentions methods for treating patients with certain CFTR mutations, such as the R117H mutation, by administering effective amounts of modulators like ivacaftor[1][5].

Key Components of the Invention

• Ivacaftor: A key modulator mentioned in the patent is ivacaftor, which is used to treat cystic fibrosis by enhancing the function of defective CFTR proteins.
• Pharmaceutical Compositions: The patent describes various pharmaceutical compositions that include these modulators, which can be administered to patients to treat or lessen the severity of diseases like cystic fibrosis[1][5].

Patent Landscape

The patent landscape surrounding US 8,354,427 is complex and involves multiple related patents and applications:

• Continuation-in-Part: This patent is a continuation-in-part of earlier applications, including U.S. patent application Ser. No. 11/165,818, filed on June 24, 2005, and U.S. Provisional Applications No. 60/582,676, No. 60/630,127, and others[1].
• Related Patents: Other patents, such as U.S. Pat. No. 7,495,103 and U.S. Pat. No. 8,324,242, also relate to modulators of ABC transporters and methods for treating cystic fibrosis. These patents collectively form a robust portfolio for Vertex Pharmaceuticals Incorporated in this therapeutic area[2][5].

Impact on Treatment of Cystic Fibrosis

The invention has significant implications for the treatment of cystic fibrosis:

• Ivacaftor and Other Modulators: The use of ivacaftor and other modulators has revolutionized the treatment of cystic fibrosis by providing a targeted therapy that can improve lung function and reduce the frequency of pulmonary exacerbations in patients with specific CFTR mutations[5].
• Personalized Medicine: The patent's focus on specific mutations aligns with the trend towards personalized medicine, where treatments are tailored to the genetic profile of the patient.

Legal and Regulatory Aspects

The patent's validity and enforcement are subject to various legal and regulatory frameworks:

• Patent Term: The patent term is extended or adjusted under 35 U.S.C. 154(b) by 377 days, reflecting the complexities of patent term adjustments[1].
• Challenges to Patent Validity: While this patent has not been specifically mentioned in the context of challenges under programs like the Covered Business Method (CBM) program, such programs provide mechanisms for challenging the validity of business method patents, which could potentially impact pharmaceutical patents if they involve business methods[3].

Conclusion

The United States Patent 8,354,427 is a pivotal invention in the field of pharmaceuticals, particularly for the treatment of cystic fibrosis. It highlights the importance of modulators of ABC transporters and their potential in addressing genetic mutations. The patent's claims and the broader patent landscape underscore the innovative efforts of Vertex Pharmaceuticals Incorporated in developing targeted therapies.

Key Takeaways

• Modulators of ABC Transporters: The patent focuses on modulators of ABC transporters, including CFTR.
• Ivacaftor: Ivacaftor is a key modulator used to treat cystic fibrosis.
• Specific Mutations: The patent addresses specific CFTR mutations, such as R117H.
• Pharmaceutical Compositions: Various pharmaceutical compositions are described for administering these modulators.
• Impact on Cystic Fibrosis: The invention has significantly improved the treatment of cystic fibrosis.

Frequently Asked Questions (FAQs)

Q: What is the primary focus of United States Patent 8,354,427? A: The primary focus is on modulators of ATP-Binding Cassette (ABC) transporters, including the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

Q: What is ivacaftor, and how is it used? A: Ivacaftor is a modulator used to treat cystic fibrosis by enhancing the function of defective CFTR proteins.

Q: Which specific mutations does the patent address? A: The patent specifically mentions methods for treating patients with the R117H mutation among other CFTR mutations.

Q: How does this patent impact the treatment of cystic fibrosis? A: The patent has revolutionized the treatment of cystic fibrosis by providing targeted therapies that improve lung function and reduce pulmonary exacerbations.

Q: What is the legal status of this patent? A: The patent is active, with its term extended or adjusted under 35 U.S.C. 154(b) by 377 days.

Cited Sources

1. United States Patent and Trademark Office, "MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS," US 8,354,427 B2, January 15, 2013.
2. United States Patent and Trademark Office, "MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS," US 11,291,662 B2, December 5, 2019.
3. Government Accountability Office, "Assessment of the Covered Business Method Patent Review Program," GAO-18-320, June 2018.
4. Google Patents, "US8354427B2 - Modulators of ATP-binding cassette transporters," Retrieved December 19, 2024.
5. Google Patents, "WO2020212898A1 - Pharmaceutical oral liquid solution of ivacaftor," Retrieved December 19, 2024.

Drugs Protected by US Patent 8,354,427

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-004	May 3, 2023		RX	Yes	No	8,354,427					TREATMENT OF CYSTIC FIBROSIS USING IVACAFTOR IN A PATIENT AGE 1 MONTH TO
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-005	May 3, 2023		RX	Yes	No	8,354,427					TREATMENT OF CYSTIC FIBROSIS USING IVACAFTOR IN A PATIENT AGE 1 MONTH TO
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,354,427					METHOD OF TREATING CYSTIC FIBROSIS
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,354,427					METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS, THE PATIENT HAVING A R117H MUTATION IN CFTR, USING N-(5-HYDROXY-2,4-DI-TERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,354,427					TREATMENT OF CYSTIC FIBROSIS USING IVACAFTOR IN A PATIENT AGE 4 MONTHS TO
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015		RX	Yes	No	8,354,427					METHOD OF TREATING CYSTIC FIBROSIS
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015		RX	Yes	No	8,354,427					TREATMENT OF CYSTIC FIBROSIS USING IVACAFTOR IN A PATIENT AGE 6 MONTHS TO

International Family Members for US Patent 8,354,427

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
European Patent Office	1773816		C300748	Netherlands
European Patent Office	1773816		CA 2015 00038	Denmark
European Patent Office	1773816		PA2015028	Lithuania
European Patent Office	1773816		C20150028 00162	Estonia
European Patent Office	1773816		1590036-8	Sweden
European Patent Office	1773816		15C0045	France
European Patent Office	1773816		92761	Luxembourg