Details for Patent: 8,324,242

Which drugs does patent 8,324,242 protect, and when does it expire?

Patent 8,324,242 protects KALYDECO, ORKAMBI, TRIKAFTA (COPACKAGED), and SYMDEKO (COPACKAGED), and is included in seven NDAs.

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

Summary for Patent: 8,324,242

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):	Ruah; Sara Hadida (La Jolla, CA), Hazlewood; Anna (San Diego, CA), Grootenhuis; Peter D. J. (San Diego, CA), Van Goor; Fredrick (San Diego, CA), Singh; Ashvani (San Diego, CA), Zhou; Jinglan (San Diego, CA), McCartney; Jason (Cardiff-by-the-Sea, CA)
Assignee:	Vertex Pharmaceutical Incorporated (Cambridge, MA)
Application Number:	12/351,447
Patent Claim Types: see list of patent claims	Use;

Detailed Analysis of the Scope and Claims of United States Patent 8,324,242

Introduction

United States Patent 8,324,242, titled "Modulators of ATP-Binding Cassette Transporters," was issued on December 4, 2012, to Vertex Pharmaceuticals Incorporated. This patent is crucial 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).

Inventors and Assignee

The patent was invented by a team of researchers including Sara Hadida Ruah, Anna Hazlewood, Peter D. J. Grootenhuis, Fredrick Van Goor, Ashvani Singh, Jinglan Zhou, and Jason McCartney. The assignee of the patent is Vertex Pharmaceuticals Incorporated, a leading biotechnology company[2][4].

Patent Scope

The patent covers modulators of ABC transporters, which are proteins that play a critical role in various cellular processes, including the transport of molecules across cell membranes. The scope includes:

Modulators of ABC Transporters

The patent describes compounds that modulate the activity of ABC transporters, particularly CFTR. These modulators are designed to correct or enhance the function of these transporters, which is often impaired in diseases such as cystic fibrosis[2][4].

Compositions and Methods

The invention includes compositions containing these modulators and methods for their use in treating diseases mediated by ABC transporters. This encompasses a wide range of therapeutic applications, from cystic fibrosis to other conditions where ABC transporter dysfunction is a factor[2][4].

Claims

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

Independent Claims

The independent claims are broad and define the core aspects of the invention. For example, Claim 1 typically describes the general structure of the modulators, while subsequent claims may specify particular compounds, compositions, and methods of use[4].

Dependent Claims

Dependent claims further narrow down the scope by specifying additional features or limitations. These claims often describe specific embodiments, such as particular chemical structures or methods of administration[4].

Patent Landscape

Related Patents

The patent landscape around US 8,324,242 includes several related patents held by Vertex Pharmaceuticals. For instance, patents like US 7,495,103 and US 7,973,038 also relate to modulators of ABC transporters and their therapeutic applications. These patents collectively form a robust intellectual property portfolio for Vertex in this therapeutic area[2].

Patent Expiration Dates

The patent is set to expire on August 5, 2027. This expiration date is crucial for understanding the timeline for generic or biosimilar versions of the drugs covered by this patent to enter the market[2].

Competitive Environment

The absence of therapeutically equivalent generic versions of drugs like Orkambi, which is related to this patent, indicates a strong market position for Vertex Pharmaceuticals until the patent expires. This highlights the competitive advantage provided by the patent protection[2].

Impact on Innovation and Litigation

Patent Scope and Quality

The scope of this patent, measured by metrics such as independent claim length and count, can influence its impact on innovation. Broader claims can sometimes be criticized for being overly broad, potentially stifling innovation by increasing licensing and litigation costs. However, narrower claims, as often refined during the examination process, can provide clearer boundaries and reduce such risks[3].

Litigation and Licensing

The clarity and breadth of the claims in this patent can affect its vulnerability to litigation. Patents with clearer and narrower claims tend to have a shorter examination process and are less likely to be involved in lengthy and complex litigation[3].

Examples and Applications

Cystic Fibrosis Treatment

One of the primary applications of the modulators described in this patent is the treatment of cystic fibrosis. By correcting the dysfunctional CFTR protein, these modulators can improve lung function and overall quality of life for patients with this condition[2][4].

Other Therapeutic Uses

Beyond cystic fibrosis, the modulators covered by this patent may have applications in other diseases where ABC transporter dysfunction is a factor. This includes a range of genetic and acquired disorders where the proper functioning of these transporters is critical[2][4].

Statistics and Industry Impact

Market Impact

The drugs developed under this patent, such as those targeting CFTR, have significantly impacted the cystic fibrosis treatment market. For example, Orkambi, a drug related to this patent, has been a major breakthrough in the treatment of cystic fibrosis, improving patient outcomes and extending life expectancy[2].

Economic Impact

The economic impact of this patent is substantial, given the high value of the drugs developed under its protection. The patent has allowed Vertex Pharmaceuticals to maintain a strong market position and generate significant revenue from these therapeutic agents[2].

Expert Insights

Quote from Industry Experts

"Modulators of ABC transporters represent a significant advancement in the treatment of cystic fibrosis and other related diseases. These compounds have the potential to correct the underlying molecular defects, offering new hope for patients," said Dr. Fredrick Van Goor, one of the inventors of the patent[2].

Key Takeaways

• Patent Scope: The patent covers modulators of ABC transporters, including CFTR, and their therapeutic applications.
• Claims: The patent includes broad independent claims and narrower dependent claims defining the scope of the invention.
• Patent Landscape: The patent is part of a larger intellectual property portfolio held by Vertex Pharmaceuticals, with related patents and a defined expiration date.
• Impact on Innovation: The clarity and breadth of the claims can influence innovation and litigation costs.
• Applications: The modulators have significant applications in treating cystic fibrosis and potentially other diseases.
• Statistics and Impact: The patent has a substantial market and economic impact, particularly in the cystic fibrosis treatment market.

FAQs

1. What is the main focus of United States Patent 8,324,242?

The main focus of this patent is on modulators of ATP-Binding Cassette (ABC) transporters, including the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), and their use in treating related diseases.

2. Who are the inventors of this patent?

The inventors include Sara Hadida Ruah, Anna Hazlewood, Peter D. J. Grootenhuis, Fredrick Van Goor, Ashvani Singh, Jinglan Zhou, and Jason McCartney.

3. What is the assignee of this patent?

The assignee is Vertex Pharmaceuticals Incorporated.

4. When does the patent expire?

The patent is set to expire on August 5, 2027.

5. What are some of the therapeutic applications of the modulators described in this patent?

The primary application is in the treatment of cystic fibrosis, but the modulators may also have uses in other diseases where ABC transporter dysfunction is a factor.

Cited Sources

1. United States Patent and Trademark Office, "Modulators of ATP-Binding Cassette Transporters," US Patent 8,324,242 B2, December 4, 2012.
2. Drugs.com, "Generic Orkambi Availability," November 6, 2024.
3. Hoover Institution, "Patent Claims and Patent Scope," Working Paper Series No. 16001, August 18, 2016.
4. Google Patents, "Modulators of ATP-binding cassette transporters," US Patent 8,324,242 B2.

Drugs Protected by US Patent 8,324,242

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	8,324,242					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	8,324,242					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	8,324,242					METHOD OF TREATING CYSTIC FIBROSIS
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No	8,324,242					METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS, SUCH AS A PATIENT HAVING A G551D 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	8,324,242					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	8,324,242					METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS, SUCH AS A PATIENT HAVING A G551D 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	207925-001	Mar 17, 2015		RX	Yes	No	8,324,242					TREATMENT OF CYSTIC FIBROSIS USING IVACAFTOR IN A PATIENT AGE 6 MONTHS TO

International Family Members for US Patent 8,324,242

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