Details for Patent: 10,022,352

« Back to Dashboard

Which drugs does patent 10,022,352 protect, and when does it expire?

Patent 10,022,352 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: 10,022,352

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 Sabina Hadida Ruah, Peter Diederik Jan Grootenhuis, Fredrick F. Van Goor, Jinglan Zhou, Brian Richard Bear, Mark Thomas Miller, Jason McCartney, Mehdi Michel Djamel Numa
Assignee:	Vertex Pharmaceuticals Inc
Application Number:	US15/078,800
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 10,022,352
Patent Claim Types: see list of patent claims	Use; Composition; Dosage form;

US Patent 10,022,352: Scope, Claims, Expiration, and Elexacaftor Patent Landscape

US Patent 10,022,352 protects cystic fibrosis treatment methods using elexacaftor, identified in the claims as Compound A, in patients carrying at least one CFTR ΔF508 mutation. It also claims solid dosage forms containing elexacaftor or a pharmaceutically acceptable salt. The patent is commercially important to Vertex Pharmaceuticals’ Trikafta and Kaftrio franchise because it covers both genotype-directed treatment and the tablet product format.

The broadest protection is divided between:

• Method claims requiring treatment of a patient with at least one ΔF508 allele.
• Combination-treatment claims covering elexacaftor with ivacaftor.
• Product claims covering solid dosage forms, including tablets and conventional excipients.
• Salt-form claims covering pharmaceutical salts of elexacaftor.

The nominal patent expiration is December 22, 2035, subject to any applicable patent-term adjustment or other term calculation shown in USPTO and FDA records.[1][2]

What drug and active ingredient does US Patent 10,022,352 protect?

US Patent 10,022,352 protects elexacaftor, also known as VX-445. The claims use the full chemical name:

(R)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide.

Elexacaftor is a CFTR corrector. It improves processing and trafficking of certain mutant CFTR proteins, particularly the F508del variant. In Trikafta and Kaftrio, elexacaftor is administered with tezacaftor and ivacaftor.

The named potentiator in claims 3 and 8 is ivacaftor:

N-(5-hydroxy-2,4-ditert-butyl-phenyl)-4-oxo-1H-quinoline-3-carboxamide.

Ivacaftor increases the probability that CFTR channels reach the cell surface and remain open. The patent therefore covers a specific combination of a CFTR corrector and CFTR potentiator, although the independent combination claims are framed as treatment methods rather than as a composition claim.

Who owns US Patent 10,022,352?

The patent is associated with Vertex Pharmaceuticals Incorporated, the developer and marketer of elexacaftor-containing CFTR modulator products. Vertex markets:

Product	Active ingredients	Principal indication
Trikafta	Elexacaftor, tezacaftor, ivacaftor	Cystic fibrosis in eligible patients aged 2 years and older
Kaftrio	Elexacaftor, tezacaftor, ivacaftor	Equivalent European product
Symdeko	Tezacaftor, ivacaftor	Earlier CFTR modulator product
Kalydeco	Ivacaftor	CFTR potentiator product

The patent’s Compound A is elexacaftor, not tezacaftor or ivacaftor. Patent analysis must therefore separate elexacaftor-specific claims from broader Trikafta portfolio patents directed to combinations, dosing, crystalline forms, manufacturing, or other formulation features.

What are the independent claims in US Patent 10,022,352?

The patent has four practical independent claim groups.

Claims 1 and 6: Treatment of patients with at least one ΔF508 allele

Claim 1 requires:

1. A patient in need of cystic fibrosis treatment.
2. Administration of an effective amount of a pharmaceutical composition.
3. The composition contains elexacaftor.
4. The patient possesses at least one CFTR ΔF508 mutation.

Claim 6 is materially similar but requires a pharmaceutically acceptable salt of elexacaftor rather than the specifically recited neutral compound.

These claims are genotype-limited. They do not cover every cystic fibrosis patient. The patient must have at least one ΔF508 allele.

The claims do not expressly require:

• Tezacaftor.
• Ivacaftor.
• A particular dose.
• A particular dosing schedule.
• A particular age.
• A particular disease severity.
• A particular formulation.
• A particular route of administration, other than what is implicit in administering the pharmaceutical composition.

The phrase “comprising” broadens the composition limitation. A product containing elexacaftor and additional active ingredients can fall within the claims if the other limitations are met.

Claims 11 and 15: Solid dosage forms

Claim 11 covers:

A solid dosage form comprising elexacaftor.

Claim 15 covers:

A solid dosage form comprising a pharmaceutically acceptable salt of elexacaftor.

These are product claims. Unlike claims 1 and 6, they do not require a cystic fibrosis indication or a patient with a ΔF508 mutation.

The claims also do not require that the solid dosage form contain ivacaftor or tezacaftor. A generic product containing elexacaftor in a solid form could present an infringement issue even if the proposed label or product design differs from the Trikafta label, assuming the product falls within the claim construction and the patent remains enforceable.

How do the dependent claims narrow the patent scope?

Claims	Limitation	Practical effect
2 and 7	Composition further comprises a CFTR potentiator	Covers combination therapy with elexacaftor and a potentiator
3 and 8	Potentiator is ivacaftor	Targets the elexacaftor-ivacaftor combination
4 and 9	ΔF508 mutation is present on both alleles	Covers homozygous F508del patients
5 and 10	Pharmaceutical composition is in solid dosage form	Links treatment claims to tablets or other solid forms
12 and 16	Solid dosage form is a tablet	Targets the principal commercial presentation
13 and 17	Fillers, lubricants, opacifying agents, or buffering agents	Adds common pharmaceutical excipient categories
14 and 18	Tablet has a film coat	Covers coated tablet presentations
19 and 20	Solid dosage form includes listed excipient categories	Repeats and confirms the excipient limitations for tablet claims

The dependent claims are commercially relevant because Trikafta is supplied as an oral tablet. However, claims 11 and 15 are already broader than the tablet claims. A tablet limitation is not necessary to infringe the independent solid-dosage-form claims if another solid dosage form satisfies the claim language.

What is the scope of the method-of-treatment claims?

The method claims focus on a defined patient population rather than on a narrow clinical endpoint.

Genotype requirement

The patient must possess at least one CFTR ΔF508 mutation. Claim 4 and claim 9 narrow the population to patients with ΔF508 mutations on both alleles.

This creates two levels of coverage:

• Heterozygous and homozygous ΔF508 patients: claims 1 and 6.
• Homozygous ΔF508 patients: claims 4 and 9.

A patient carrying one ΔF508 allele and another non-ΔF508 mutation can fall within claims 1 and 6 if the remaining limitations are met.

Active-ingredient requirement

Claims 1 through 10 require elexacaftor or its pharmaceutically acceptable salt. A product containing only tezacaftor and ivacaftor would not meet these claims.

Combination requirement

Claims 2, 3, 7, and 8 add a potentiator requirement. Claims 3 and 8 specifically identify ivacaftor. These claims are particularly relevant to a three-component CFTR regimen when elexacaftor, tezacaftor, and ivacaftor are administered together, because the composition can contain additional active ingredients under the “comprising” language.

Dosage-form requirement

Claims 5 and 10 require a solid dosage form. A liquid or other non-solid formulation may avoid these dependent claims but would remain potentially relevant to the broader method claims 1 and 6.

What formulations are protected by US Patent 10,022,352?

The formulation claims cover solid dosage forms containing elexacaftor or a pharmaceutically acceptable salt. The patent does not limit the independent solid-dosage-form claims to a particular:

• Tablet weight.
• Elexacaftor concentration.
• Release profile.
• Particle size.
• Crystalline form.
• Excipient identity.
• Manufacturing process.
• Coating thickness.
• Dissolution specification.

The dependent claims identify common excipient categories:

• Fillers.
• Lubricants.
• Opacifying agents.
• Buffering agents.
• Film coats.

The use of “comprising” means the listed excipients are not necessarily exclusive. A tablet with additional binders, disintegrants, glidants, colorants, stabilizers, or other excipients can remain within the claim scope.

Salt coverage

Claims 6 through 10 and 15 through 18 expressly cover pharmaceutically acceptable salts of elexacaftor. This limits a potential design-around based solely on converting the free compound into a salt.

The claims do not identify a single salt species. Their breadth depends on whether the proposed salt is pharmaceutically acceptable and whether the product otherwise meets the applicable composition or method claim.

What is the Orange Book status of US Patent 10,022,352?

US Patent 10,022,352 is associated with Vertex’s elexacaftor-containing cystic fibrosis product portfolio and has been identified in connection with Trikafta patent protection in FDA patent listings.[2]

The Orange Book is important because a listed patent can trigger statutory notice and litigation consequences when an ANDA applicant files a Paragraph IV certification. The listing does not itself establish that every commercial presentation infringes every claim. Claim scope, product composition, labeling, and patent enforceability remain separate questions.

For an ANDA applicant, the key exposure points are:

• A proposed product containing elexacaftor.
• A tablet or other solid dosage form.
• A label directed to cystic fibrosis patients with the relevant CFTR genotype.
• A combination product containing ivacaftor.
• A proposed label that encourages use in the claimed ΔF508 population.

When does US Patent 10,022,352 lose exclusivity?

The nominal expiration date is December 22, 2035.[1][2] The practical launch date for a generic may be later or earlier depending on:

• Paragraph IV litigation.
• A 30-month stay under Hatch-Waxman.
• Settlement terms.
• Patent-term adjustment.
• Pediatric exclusivity.
• Other unexpired patents listed for Trikafta.
• Regulatory exclusivity applicable to the relevant active ingredient or product.

The patent should not be viewed in isolation. Even if a generic applicant defeats or avoids US Patent 10,022,352, other Vertex patents may continue to block launch.

Regulatory exclusivity

Trikafta received FDA approval on October 21, 2019. Elexacaftor was a new active ingredient in the United States, but the regulatory exclusivity analysis for a combination product is separate from patent term. FDA approval and exclusivity information must be evaluated through the Orange Book and FDA approval records.[2][3]

The principal commercial barrier is expected to be patent protection rather than the five-year new-chemical-entity period, which does not extend to the 2035 patent expiration date.

How strong is the patent estate around elexacaftor and Trikafta?

The estate is stronger than US Patent 10,022,352 alone suggests because Vertex has used multiple patent categories.

Patent category	Protected subject matter	Generic risk
Compound patents	Elexacaftor chemical structure	High barrier if valid and unexpired
Treatment patents	CFTR genotype, patient population, and dosing	Label and induced-infringement risk
Combination patents	Elexacaftor with tezacaftor and ivacaftor	Relevant to fixed-dose and co-packaged products
Formulation patents	Tablets, salts, excipients, release characteristics	Product-specific infringement risk
Solid-form patents	Crystalline or amorphous forms	Potential manufacturing and characterization barrier
Manufacturing patents	Intermediates, processes, purification	API supplier and CMO risk
Pediatric or dosing patents	Age, weight, and regimen limitations	Narrower but commercially useful protection

US Patent 10,022,352 is strongest against a conventional oral solid product that contains elexacaftor and is labeled for patients with at least one F508del mutation. Its treatment claims are narrower than a compound patent because they depend on the patient genotype and treatment use. Its solid-dosage-form claims are broader in product format because they do not require a cystic fibrosis indication.

What Paragraph IV challenges and generic launch risks exist?

A generic applicant seeking approval for an elexacaftor-containing product would likely need to address listed patents through one or more of the following certifications:

• Paragraph III certification, accepting delay until patent expiration.
• Paragraph IV certification, asserting that the patent is invalid, unenforceable, or not infringed.
• Section VIII statement, where a patent is not relevant to the proposed label.

US Patent 10,022,352 creates several Paragraph IV theories.

Noninfringement strategies

A challenger could argue that:

• The proposed product is not a solid dosage form.
• The product does not contain elexacaftor as claimed.
• The salt is outside the scope of “pharmaceutically acceptable salt.”
• The proposed labeling does not direct treatment of patients with a ΔF508 mutation.
• The product does not contain ivacaftor, avoiding the dependent combination claims.
• The proposed product is not intended for the claimed method.

The solid-dosage-form claims make a formulation-based noninfringement position more difficult for a conventional tablet product.

Invalidity strategies

Potential invalidity issues would ordinarily include:

• Lack of written description for the full salt or dosage-form scope.
• Enablement of the full range of solid dosage forms and salts.
• Obviousness based on earlier CFTR corrector and potentiator disclosures.
• Claim construction disputes over “pharmaceutical composition,” “solid dosage form,” and “pharmaceutically acceptable salt.”
• Double-patenting issues involving related elexacaftor patents.
• Priority and inventorship issues, if supported by the prosecution record.

The method claims may face a different obviousness analysis from the formulation claims. A prior-art reference disclosing elexacaftor and CFTR treatment may not automatically establish obviousness of the genotype-specific treatment limitation or the claimed tablet composition.

What litigation affects US Patent 10,022,352?

The relevant litigation risk is primarily Hatch-Waxman litigation arising from ANDA filings for elexacaftor-containing products. Vertex has historically enforced its CFTR portfolio against generic applicants, and litigation can involve multiple patents in the Trikafta listing rather than a single patent.

A case-specific assessment requires separating:

1. Whether US Patent 10,022,352 was asserted.
2. Whether the patent was dismissed, stayed, settled, or adjudicated.
3. Whether a settlement permits an authorized generic or a date-certain launch.
4. Whether the patent remains listed for the relevant NDA.
5. Whether any terminal disclaimer or patent-term adjustment changes the expiration calculation.

No biosimilar pathway applies. Elexacaftor is a chemically synthesized small molecule, so competitors would generally pursue an ANDA rather than a 351(k) biosimilar application.

Are there licensing deals affecting this patent?

Vertex’s cystic fibrosis franchise originated from a broader development and licensing relationship involving the Cystic Fibrosis Foundation. The Foundation supported early discovery and development work and entered agreements connected with Vertex’s CFTR program.[4]

Those arrangements are commercially relevant to the historical development of ivacaftor and related CFTR modulators. They do not, by themselves, determine whether a third party may practice US Patent 10,022,352. Patent ownership, license scope, sublicensing rights, and any field or territory restrictions must be assessed from the underlying agreements.

How does US Patent 10,022,352 compare with other Trikafta patents?

US Patent 10,022,352 has a mixed claim profile.

Issue	US 10,022,352	Typical compound patent	Typical formulation patent
Claims elexacaftor structure directly	No, based on supplied claims	Yes	Usually no
Covers CFTR genotype	Yes	Usually no	Usually no
Covers elexacaftor plus ivacaftor	Yes, by method claims	Sometimes	Sometimes
Covers tablets	Yes	No	Yes
Covers salts	Yes	Sometimes	Yes
Requires cystic fibrosis treatment	Method claims only	No	Usually no
Vulnerable to label carve-out	More vulnerable for method claims	Less vulnerable	Product-specific
Relevant to Trikafta tablets	Yes	Yes if product patent	Yes

The patent’s main value is its combination of patient-selection claims and broad solid-dosage-form claims. A challenger that avoids the genotype-specific method claims may still face the independent product claims for a tablet containing elexacaftor.

What is the commercial exposure if the patent is successfully challenged?

Trikafta is Vertex’s principal cystic fibrosis product and one of the company’s largest revenue contributors. A successful early generic launch could affect:

• US net product revenue.
• Pricing and payer contracting.
• Patient conversion to generic therapy.
• International reference pricing.
• Vertex’s cystic fibrosis cash flows.
• The value of follow-on patent litigation and settlements.

The economic impact would depend on whether the generic is authorized for the full Trikafta population, whether it contains all three active ingredients, whether launch is at risk, and whether other patents remain enforceable.

Because the patent covers elexacaftor-containing solid dosage forms, a successful challenge could have consequences beyond one narrow genotype indication if the generic product itself satisfies claims 11 or 15.

Key Takeaways

• US Patent 10,022,352 protects methods using elexacaftor in cystic fibrosis patients with at least one CFTR ΔF508 mutation.
• Claims 2, 3, 7, and 8 address combinations with a CFTR potentiator, specifically ivacaftor.
• Claims 11 through 20 cover solid dosage forms, tablets, pharmaceutical salts, excipients, and film-coated tablets.
• The independent solid-dosage-form claims do not require a cystic fibrosis indication.
• The patent is commercially relevant to Trikafta and Kaftrio.
• The nominal expiration date is December 22, 2035, subject to the official USPTO and Orange Book term records.
• Elexacaftor is a small molecule, so generic challengers would use the ANDA pathway, not the biosimilar pathway.
• A Paragraph IV challenger would face both method-of-use and product-formulation infringement theories.
• The patent is one component of a larger Vertex patent estate covering compounds, combinations, formulations, solid forms, dosing, and manufacturing.
• A successful challenge to this patent would not necessarily establish immediate market entry if other unexpired Trikafta patents remain enforceable.

FAQs

Does US Patent 10,022,352 cover ivacaftor by itself?

No. The supplied claims require elexacaftor or a pharmaceutically acceptable salt of elexacaftor. Ivacaftor appears as an additional potentiator in dependent claims.

Does the patent cover patients without an F508del mutation?

The treatment claims supplied here require at least one CFTR ΔF508 mutation. They do not cover treatment of patients who lack that mutation. The solid-dosage-form claims do not contain a patient-genotype limitation.

Can a liquid elexacaftor formulation avoid US Patent 10,022,352?

A liquid formulation could avoid the solid-dosage-form claims if it does not meet the claim construction for a solid dosage form. It could still implicate the broader treatment claims if used to treat a patient with at least one ΔF508 mutation.

Are Trikafta tablets protected even if they contain tezacaftor and ivacaftor?

Yes, the “comprising” language permits additional ingredients. A tablet containing elexacaftor together with tezacaftor and ivacaftor can still fall within a claim requiring a solid dosage form comprising elexacaftor.

Is US Patent 10,022,352 a biologic patent?

No. Elexacaftor is a chemically synthesized small molecule. The relevant approval pathway for a competing product is generally an ANDA, not a 351(k) biosimilar application.

References

1. United States Patent and Trademark Office. (2018). US Patent No. 10,022,352, methods of treating cystic fibrosis. USPTO Patent Center. https://patentcenter.uspto.gov/
2. U.S. Food and Drug Administration. (n.d.). Approved drug products with therapeutic equivalence evaluations: Orange Book. https://www.accessdata.fda.gov/scripts/cder/ob/
3. U.S. Food and Drug Administration. (2019). TRIKAFTA prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/
4. Cystic Fibrosis Foundation. (n.d.). CF Foundation and Vertex Pharmaceuticals collaboration and cystic fibrosis drug development. https://www.cff.org/

Drugs Protected by US Patent 10,022,352

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	GRANULE;ORAL	217660-001	Apr 26, 2023		RX	Yes	No	10,022,352		Y			TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO
Vertex Pharms Inc	TRIKAFTA (COPACKAGED)	elexacaftor, ivacaftor, tezacaftor; ivacaftor	GRANULE;ORAL	217660-002	Apr 26, 2023		RX	Yes	Yes	10,022,352		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	10,022,352		Y			TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE WITH AN EFFECTIVE AMOUNT OF A PHARMACEUTICAL COMPOSITION COMPRISING VANZACAFTOR, TEZACAFTOR, AND DEUTIVACAFTOR

International Family Members for US Patent 10,022,352

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Australia	2007235260
Brazil	PI0710965
Canada	2648719