Details for Patent: 8,741,933

Which drugs does patent 8,741,933 protect, and when does it expire?

Patent 8,741,933 protects ORKAMBI and is included in two NDAs.

This patent has ninety-one patent family members in twenty-five countries.

Summary for Patent: 8,741,933

Title:	Modulators of ATP-binding cassette transporters
Abstract:	Compounds of the present invention, and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator ("CFTR"). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.
Inventor(s):	Hadida Ruah; Sara S. (La Jolla, CA), Hamilton; Matthew (Hackettstown, NJ), Miller; Mark (San Diego, CA), Grootenhuis; Peter D. J. (San Diego, CA), Bear; Brian (Carlsbad, CA), McCartney; Jason (Cardiff by the Sea, CA), Zhou; Jinglan (San Diego, CA)
Assignee:	Vertex Pharmaceuticals Incorporated (Boston, MA)
Application Number:	13/871,356
Patent Claim Types: see list of patent claims	Use; Composition;

Understanding the Scope and Claims of United States Patent 8,741,933

Introduction

United States Patent 8,741,933, assigned to Vertex Pharmaceuticals Incorporated, is a significant patent in the field of pharmaceuticals, particularly relating to the treatment of cystic fibrosis. This analysis will delve into the scope, claims, and the broader patent landscape surrounding this patent.

Background of the Patent

The patent in question, 8,741,933, is part of a series of patents related to the drug ORKAMBI (lumacaftor and ivacaftor), which is used to treat cystic fibrosis. Cystic fibrosis is a genetic disorder that affects the lungs, pancreas, and other organs, and ORKAMBI is designed to modulate the CFTR (cystic fibrosis transmembrane conductance regulator) protein, which is defective in patients with this condition[2][4].

Scope of the Patent

The scope of the patent encompasses compounds and methods for treating diseases associated with defective ABC (ATP-binding cassette) transporters, including CFTR. The invention includes specific chemical compounds that act as modulators of these transporters, thereby improving the function of the defective CFTR protein in cystic fibrosis patients.

Claims of the Patent

The patent includes several claims that define the scope of protection afforded by the patent. Here are some key aspects of the claims:

• Compound Claims: The patent claims specific chemical compounds that are designed to modulate the CFTR protein. These compounds are detailed in the specification and are critical for the therapeutic effect of the drug[4].
• Method Claims: The patent also includes method claims that describe how these compounds are used to treat cystic fibrosis. This includes the administration of the compounds and the therapeutic outcomes expected[4].
• Composition Claims: Additionally, the patent claims pharmaceutical compositions that include these compounds, along with other ingredients that enhance their delivery and efficacy[4].

Patent Landscape

To understand the broader patent landscape, it is essential to consider several factors:

• Technology Field: The patent falls under the "Drugs and Medical" technology field, one of the six broad categories defined by the USPTO's NBER classification scheme[1].
• Continuation Applications: The patent is part of a family of patents that includes continuation applications. This is common in pharmaceutical patents where multiple applications are filed to cover different aspects of the invention, such as new compounds, methods of use, or formulations[1][2].
• Regulatory Review: The regulatory review period for ORKAMBI, the drug associated with this patent, was extensive, lasting 2,785 days. This highlights the rigorous process pharmaceuticals undergo before approval[2].

Allowance Rates and Patent Statistics

Understanding the probability of receiving a US patent can provide context for the significance of this patent. The USPTO calculates several measures of patent allowance rates, including the first-action allowance rate, progenitor allowance rate, and family allowance rate. For pharmaceutical patents, these rates can vary significantly based on the complexity of the invention and the scrutiny applied during the examination process[1].

Legal and Judicial Aspects

Patent claims must be precise enough to afford clear notice of what is claimed, as mandated by 35 U.S.C. § 112(b). The Federal Circuit has emphasized that claims must inform those skilled in the art about the scope of the invention with reasonable certainty, while recognizing that absolute precision is unattainable[5].

Examples and Case Studies

The patent for ORKAMBI is a prime example of how complex pharmaceutical inventions are protected. Vertex Pharmaceuticals' efforts in developing and patenting this drug involved extensive research and multiple patent applications. This process is reflective of the broader strategy in the pharmaceutical industry to secure comprehensive patent protection for innovative treatments.

Industry Expert Insights

Industry experts often highlight the importance of robust patent protection in the pharmaceutical sector. For instance, the development of ORKAMBI and its associated patents demonstrate the significant investment and innovation required to bring new treatments to market. As noted by industry experts, strong patent protection is crucial for incentivizing such investments[1].

Illustrative Statistics

• Patent Allowance Rates: The allowance rate for pharmaceutical patents can be lower compared to other technology fields due to the stringent examination process. For example, the progenitor allowance rate for pharmaceutical patents might be around 50-60%, indicating the challenging nature of obtaining patent approval in this field[1].
• Regulatory Approval Time: The regulatory review period for ORKAMBI, at 2,785 days, underscores the lengthy and rigorous process involved in approving new drugs[2].

Key Takeaways

• Scope and Claims: The patent 8,741,933 covers specific compounds and methods for treating cystic fibrosis by modulating the CFTR protein.
• Patent Landscape: The patent is part of a broader family of patents related to ORKAMBI and falls under the "Drugs and Medical" technology field.
• Legal and Regulatory Aspects: The patent must meet the legal standards for claim definiteness and has undergone extensive regulatory review.
• Industry Significance: The patent reflects the significant investment and innovation in the pharmaceutical industry, particularly in developing treatments for complex genetic disorders.

FAQs

Q: What is the main subject of United States Patent 8,741,933? A: The main subject is the compounds and methods for treating cystic fibrosis by modulating the CFTR protein.

Q: Who is the assignee of this patent? A: The assignee is Vertex Pharmaceuticals Incorporated.

Q: What is the regulatory review period for ORKAMBI? A: The regulatory review period for ORKAMBI was 2,785 days.

Q: Why is patent protection important in the pharmaceutical industry? A: Patent protection is crucial for incentivizing investment in research and development of new treatments.

Q: What are the key claims in this patent? A: The key claims include compound claims, method claims, and composition claims related to the modulation of the CFTR protein.

Cited Sources

1. Carley, M., & Hegde, D. (n.d.). What Is the Probability of Receiving a US Patent?. Retrieved from https://yjolt.org/sites/default/files/carley_hegde_marco-what_is_the_probability_of_receiving_a_us_patent_0.pdf
2. United States Patent and Trademark Office. (2017). Acting Director United States Patent and Trademark Office Mail Stop Hatch-Waxman PTE P.O. Box 1450 Alexandria, VA 22313-1450. Retrieved from https://downloads.regulations.gov/FDA-2016-E-1301-0004/attachment_1.pdf
3. United States Patent and Trademark Office. (2017). Patent Claims Research Dataset. Retrieved from https://www.uspto.gov/ip-policy/economic-research/research-datasets/patent-claims-research-dataset
4. United States Patent and Trademark Office. (2019). United States Patent - googleapis.com. Retrieved from https://patentimages.storage.googleapis.com/0b/09/83/6ac173df846860/US10987348.pdf
5. United States Court of Appeals for the Federal Circuit. (2022). NATURE SIMULATION SYSTEMS INC. v. AUTODESK, INC.. Retrieved from https://cafc.uscourts.gov/opinions-orders/20-2257.OPINION.10-17-2022_2019495.pdf

Drugs Protected by US Patent 8,741,933

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	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-003	Sep 2, 2022		RX	Yes	No	8,741,933					TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	8,741,933					TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes	8,741,933					TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-002	Sep 28, 2016		RX	Yes	No	8,741,933					METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND LUMACAFTOR

International Family Members for US Patent 8,741,933

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
European Patent Office	2404919		PA2016015	Lithuania
European Patent Office	2404919		93073	Luxembourg
European Patent Office	2404919		CA 2016 00019	Denmark
European Patent Office	2395002		300812	Netherlands