Details for Patent: 11,639,347

Which drugs does patent 11,639,347 protect, and when does it expire?

Patent 11,639,347 protects TRIKAFTA (COPACKAGED), ALYFTREK, and SYMDEKO (COPACKAGED), and is included in four NDAs.

This patent has fifty-two patent family members in twenty-one countries.

Summary for Patent: 11,639,347

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):	Sara S. Hadida Ruah, Peter D. J. Grootenhuis, Fredrick Van Goor, Jinglan Zhou, Brian Bear, Mark T. Miller, Jason McCartney, Mehdi Michel Jamel Numa, Xiaoqing Yang
Assignee:	Vertex Pharmaceuticals Inc
Application Number:	US17/181,931
Patent Claim Types: see list of patent claims	Use; Composition;

United States Patent 11,639,347: Scope, Claim Architecture, and US Landscape Implications

US Patent 11,639,347 is directed to a family of substituted compounds defined by broad, structure-driven Markush frameworks (Forms IIc and IId), plus pharmaceutical compositions and cystic fibrosis treatment methods. The claims are drafted to capture (i) wide latitude in substituent substitution patterns on multiple variable segments, (ii) multiple allowed “replacement” motifs within C1-6 linker fragments, and (iii) broad salt coverage. Downstream coverage extends through composition claims and therapeutic-use claims for cystic fibrosis, including narrower dependent coverage keyed to specific R-group instantiations.

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What does the core claim coverage include? (Compound claims 1-5)

Claim 1: “Formula IIc” compound family with extensive Markush substitution

Claim 1 recites a compound of formula IIc (or a pharmaceutically acceptable salt). Scope is controlled by multiple layers of definitions:

Key variables

• R1 is —ZAR4

  • Each ZA independently is either:
  • a bond, or
  • an optionally substituted branched or straight C1-6 aliphatic chain, where up to two carbon units can be replaced by a long list of heteroatom or heterogroup inserts:
    • Carbon replacements include: —CO—, —CS—, —CONRA—, —CONRANRA—, —CO2—, —OCO—, —NRACO2, —O—, —NRACONRA—, —OCONRA—, —NRANRA—, —NRACO—, —S—, —SO—, —SO2—, —NRA—, —SO2NRA—, —NRASO2, —NRASO2NRA—
  • This gives claim reach into multiple heteroatom-rich “alkyl-to-heteroalkyl” substitutions without needing a separate claim per motif.
  • Each R4 is independently selected from:
  • RA, halo, —OH, —NH2, —NO2, —CN, —OCF3
  • Each RA is independently selected from:
  • hydrogen
  • optionally substituted aliphatic / cycloaliphatic / heterocycloaliphatic
  • optionally substituted aryl / heteroaryl

• R2 is —ZBR5

  • Each ZB independently is either:
  • a bond, or
  • an optionally substituted branched/straight C1-6 aliphatic chain, with up to two carbon units replaced by an extensive set analogous to Claim 1:
    • —CO—, —CS—, —CONRB—, —CONRBNRB—, —CO2—, —OCO—, —NRBCO2, —O—, —NRBCONRB—, —OCONRB—, —NRBNRB—, —NRBCO—, —S—, —SO—, —SO2—, —NRB—, —SO2NRB—, —NRBSO2, —NRBSO2NRB—
  • Each R5 is independently selected from:
  • RB, halogen, —OH, —NH2, —NO2, —CN, CF3, —OCF3
  • Each RB mirrors RA but with “RB” label:
  • hydrogen or optionally substituted aliphatic / cycloaliphatic / heterocycloaliphatic / aryl / heteroaryl

• Or topology closure on R2:

  • “any two adjacent R2 groups, together with the atoms to which they are attached, form an optionally substituted carbocycle or heterocycle”
  • This permits ring closure across adjacent R2 sites, widening chemical space beyond acyclic R2 substituents.

• R3 and R′3:

  • each is —ZCR6
  • ZC is analogous to ZA/ZB:
  • bond or optionally substituted C1-6 aliphatic chain with up to two carbon units replaced by a further list (analogous to above) including:
    • —CO—, —CS—, —CONRC—, —CONRCNRC—, —CO2—, —OCO—, —NRCCO2, —O—, —NRCCONRC—, —OCONRC—, —NRCNRC—, —NRCCO—, —S—, —SO—, —SO2—, —NRC—, —SO2NRC—, —NRCSO2, —NRCSO2NRC—
  • R6 is selected from:
  • RC, halogen, —OH, —NH2, —NO2, —CN, —OCF3
  • RC mirrors the “hydrogen + optionally substituted aryl/heteroaryl and cyclic/acyclic aliphatic variants”.

• Substitution limits:

  • n = 1–3
  • p = 0–3

Functional impact on scope

Claim 1 is a “broad genus” claim:

• It does not restrict biological mechanism.
• It allows multiple heteroatom insertion patterns within short linkers (C1-6 with up to two carbon replacements), enabling numerous isosteric analogs.
• It permits ring-formation at adjacent R2 sites, adding further structural coverage.

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Claim 2: “Formula IId” with tighter element: R2 dimer cyclization/structure constraint

Claim 2 recites a compound of formula IId (or salt). Compared with Claim 1, Claim 2 inserts a categorical restriction:

• “both R2 groups, together with the atoms to which they are attached, form a group selected from: …”
• The remaining text is truncated in the provided claims excerpt, but the structure clearly shifts from Claim 1’s independently defined R2 side chains toward a restricted R2 together-forming scaffold.

The excerpt also indicates:

• R′3 and R3 definitions are present (later partially shown).
• Claim 2 appears to be a subset of the broader chemotype space, typically used to capture preferred embodiments with defined ring or motif formation.

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Claims 3-5: dependent narrowing to specific R2 and R′3 constructs

• Claim 3 narrows Claim 2 by selecting a specific “group” for the R2-constrained formation (text truncated in the excerpt, but Claim 3 depends on Claim 2).
• Claim 4 adds a further constraint on R′3, stating R′3 is hydrogen or a specific group defined using R31, R32, R33, and L:
  • R31:
  • hydrogen or a C1-2 aliphatic optionally substituted with 1-3 halogen or —OH
  • R32 is —L—R33
  • L is one of:
  • bond, —CH2—, —CH2O—, —CH2NHS(O)2, —CH2C(O)—, —CH2NHC(O)—, —CH2NH—
  • R33 is:
  • hydrogen or C1-2 aliphatic / cycloaliphatic / heterocycloaliphatic / heteroaryl, each optionally substituted with —OH, —NH2, or —CN
• Claim 5 is narrower than Claim 4:
  • R31 = hydrogen
  • R32 is C1-2 aliphatic optionally substituted with one of —OH, —NH2, or —CN

Scope implication: these dependents lock specific substitution patterns likely corresponding to commercially relevant or synthesis-plausible embodiments. They also provide fallback coverage against partial validity challenges.

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How broad are the formulation and combination claims? (6-9)

Claim 6: pharmaceutical composition

• “A pharmaceutical composition comprising a compound (or salt) according to any one of claims 1-4, and a pharmaceutically acceptable carrier, adjuvant, or vehicle.”

This claim is broad across the entire compound genus and its narrowed subsets.

Claim 7: combination therapy

• Adds: “further comprises one or more additional therapeutic agents.”

This is a standard combination claim that can cover co-administration regimens, subject to practical evidence.

Claims 8-9: compositions keyed to Claim 5

• Claim 8 restricts the compound set to Claim 5 specifically.
• Claim 9 again adds optional additional therapeutic agents.

Landscape implication: There are two “composition tiers”:

• a genus-tier composition (claims 1-4),
• and an embodiment-tier composition (claim 5).

This tiering often supports enforcement by capturing both broad and preferred structures in separate claims.

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What is the therapeutic-use claim scope? (10-11)

Claim 10: cystic fibrosis treatment (any of claims 1-4)

• “A method of treating or lessening the severity of cystic fibrosis in a patient” by administering an effective amount of a compound/salt according to any one of claims 1-4.

Coverage elements:

• Disease: cystic fibrosis
• Action: “treating or lessening severity”
• Administration: “administering to a patient an effective amount”
• Chemical scope: claims 1-4

Claim 11: cystic fibrosis treatment (keyed to Claim 5)

• Same disease and method structure, but limits the compound set to Claim 5 only.

Scope implication: Claim 10 provides broad therapeutic-use coverage at the genus level; Claim 11 is a fallback narrower embodiment.

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Where are the legal pressure points in claim drafting?

1) The genus is extremely broad in “hetero-replacement” motifs

Claims 1’s allowance to replace up to two carbon units within C1-6 chains with multiple heteroatomic constructs is a major breadth lever:

• It increases the number of included chemical structures without needing to recite each one.
• It also makes invalidity arguments dependent on whether prior art discloses these multi-replacement variants specifically, not merely the base scaffold.

2) Ring closure permissiveness via adjacent R2

The statement that adjacent R2 groups can form an optionally substituted carbocycle or heterocycle adds further breadth beyond simple substitution:

• This can pull in structurally different but functionally similar analogs.
• It may complicate novelty assessments if prior art includes intermediate ring-closure chemotypes.

3) Dependent claims lock “preferred” R′3 and linker L motifs

Claims 4-5 provide specific linker and substitution patterns:

• L restricted to a finite set (bond, CH2, CH2O, CH2NHS(O)2, CH2C(O), CH2NHC(O), CH2NH)
• R31 restricted to hydrogen or C1-2 aliphatic with selected substitution types
• These create clearer subject matter that can be easier to map to prior art if those linker motifs are already known in analogous cystic fibrosis chemotypes.

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US patent landscape: scope-to-landscape translation for freedom-to-operate

The excerpt you provided contains only claim text. Without the patent’s bibliographic metadata (assignee, filing date, application number, priority dates) and without the rest of the specification, a complete and accurate cross-patent landscape cannot be produced from the record you supplied.

So the only landscape-safe conclusions available from the claim architecture itself are structural: what kinds of earlier patents are most likely to overlap based on how the claims are written.

Overlap risk categories implied by the claim language

Given the claims:

• Method is for cystic fibrosis
• Compound genus is defined by multi-part Markush substituent sets
• Linkers allow common medicinal chemistry motifs:
  • heteroatoms (O, S),
  • sulfonamides (CH2NHS(O)2),
  • amides/ketones (CH2C(O), CH2NHC(O), CH2NH—),
  • nucleophilic substituents including —OH, —NH2, —CN
  • halogen and specific fluorinated groups (CF3, OCF3)

The highest overlap risk in earlier US portfolios typically comes from patents that:

1. Disclose cystic fibrosis-targeting small molecules with the same or closely related pharmacophore scaffold, and use substituent frameworks containing:
   • CF3/OCF3/halo/CN/OH/NH2
   • sulfonamide, amide, and ether-like linkers
2. Disclose the same formula family boundaries (n = 1–3; p = 0–3) using related Markush variations, especially if prior art includes “up to two carbon replacements” within short chains.
3. Claim pharmaceutical compositions and treatment of cystic fibrosis for the same compound classes.

Enforcement leverage from dependent claim structure

Even if a genus claim is challenged, dependent claims 4-5 and composition and method claims 11 can still be asserted if:

• a specific R′3/L/R32/R33 embodiment remains novel and non-obvious; and
• the earlier art does not disclose the exact dependent configuration.

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Practical claim mapping: how to read coverage in FTO terms

What a competitor design needs to avoid

To design around Claim 1 broadly, a competitor would need to:

• Change the core chemotype such that R1/R2/R3 definitions no longer fit the formula IIc constraints, including:
  • ZA/ZB/ZC replacement rules,
  • allowed R4/R5/R6 substituent sets,
  • and the n/p limits governing substitution counts.
• Or alter the structure so that adjacency of R2 sites does not permit ring formation as claimed.

What is harder to design around

• The combination of broad allowed substituent classes and broad hetero-replacement options means small modifications often remain within Markush coverage unless they break the claimed variables or scaffold identity.

What to focus on for design around

• The most targeted safe-space efforts typically occur at:
  • the linker identity corresponding to the L set in dependent claim 4,
  • the R′3 architecture constrained in claim 4-5,
  • and whether the compound can plausibly be argued to fall outside “formula IId” or “formula IIc” boundaries by altering the structural core rather than just adding substituents.

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Key Takeaways

1. Claim 1 is a broad genus over formula IIc with extensive Markush latitude: ZA/ZB/ZC are bond or substituted C1-6 chains with up to two carbon units replaced by many heteroatomic motifs, and R1/R2/R3 substituents allow broad RA/RB/RC and halo/CF3/OCF3/OH/NH2/CN/NO2 sets.
2. Claims 2-5 narrow into formula IId with R2 together-forming constraints and then further limit R′3 via specific R31/R32/R33 and linker L selections (finite L set; constrained substituent classes).
3. Claims 6-9 cover pharmaceutical compositions for the compound genus (claims 1-4) and a preferred subset (claim 5), including optional addition of other therapeutic agents.
4. Claims 10-11 provide cystic fibrosis therapeutic-use coverage at the genus level (claims 1-4) and fallback level (claim 5).
5. From an FTO perspective, the biggest structural overlap risk is with earlier cystic fibrosis chemotype patents that use similar medicinal chemistry motifs (CF3/OCF3, sulfonamides, amides/ketones, short-chain hetero-replacement logic). Design around most often requires changing the chemotype boundaries, not just minor substituent tweaks.

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FAQs

1) Is the patent limited to a specific mechanism of action?

No. The provided claims define chemical structure (formula-based Markush) and therapeutic use, not a specific MOA term.

2) Do the claims cover salts?

Yes. Claims 1-5 explicitly cover pharmaceutically acceptable salts.

3) Are combination therapies included?

Yes. Claims 7 and 9 add “one or more additional therapeutic agents” to the composition.

4) Which claims provide the narrowest cystic fibrosis method coverage?

Claim 11, because it limits the administered compound to claim 5.

5) What is the most structurally specific claim element in the excerpt?

The dependent claim 4 linker definition L is restricted to a finite set (bond, CH2, CH2O, CH2NHS(O)2, CH2C(O), CH2NHC(O), CH2NH).

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References (Cited Sources)

1. User-provided claim text for US Patent 11,639,347 (claims 1-11).

Drugs Protected by US Patent 11,639,347

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	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-001	Apr 26, 2023		RX	Yes	No			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULES;ORAL	217660-002	Apr 26, 2023		RX	Yes	Yes			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO
Vertex Pharms Inc	ALYFTREK	deutivacaftor; tezacaftor; vanzacaftor calcium	TABLET;ORAL	218730-001	Dec 20, 2024		RX	Yes	No			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION OR ANOTHER RESPONSIVE MUTATION IN THE CFTR GENE WITH AN EFFECTIVE AMOUNT OF VANZACAFTOR, TEZACAFTOR, AND DEUTIVACAFTOR
Vertex Pharms Inc	ALYFTREK	deutivacaftor; tezacaftor; vanzacaftor calcium	TABLET;ORAL	218730-002	Dec 20, 2024		RX	Yes	Yes			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION OR ANOTHER RESPONSIVE MUTATION IN THE CFTR GENE WITH AN EFFECTIVE AMOUNT OF VANZACAFTOR, TEZACAFTOR, AND DEUTIVACAFTOR
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-002	Jun 21, 2019		RX	Yes	No			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR
Vertex Pharms Inc	SYMDEKO (COPACKAGED)	ivacaftor; ivacaftor, tezacaftor	TABLET;ORAL	210491-001	Feb 12, 2018		RX	Yes	Yes			Y	Y		TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR

International Family Members for US Patent 11,639,347

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Australia	2007235260
Brazil	PI0710965
Canada	2648719
Canada	2869945
China	101460489
China	103254177