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KALYDECO Drug Patent Profile

When do Kalydeco patents expire, and what generic alternatives are available?

Kalydeco is a drug marketed by Vertex Pharms Inc and Vertex Pharms and is included in two NDAs. There are thirteen patents protecting this drug and two Paragraph IV challenges.

This drug has two hundred and fifty-five patent family members in thirty-three countries.

The generic ingredient in KALYDECO is ivacaftor. There are three drug master file entries for this compound. One supplier is listed for this compound. Additional details are available on the ivacaftor profile page.

DrugPatentWatch^® Generic Entry Outlook for Kalydeco

Kalydeco was eligible for patent challenges on January 31, 2016.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be August 13, 2029. This may change due to patent challenges or generic licensing.

There has been one patent litigation case involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

There is one tentative approval for the generic drug (ivacaftor), which indicates the potential for near-term generic launch.

Indicators of Generic Entry

Summary for KALYDECO

International Patents:	255
US Patents:	13
Applicants:	2
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	104
Clinical Trials:	26
Patent Applications:	831
Drug Prices:	Drug price information for KALYDECO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for KALYDECO
What excipients (inactive ingredients) are in KALYDECO?	KALYDECO excipients list
DailyMed Link:	KALYDECO at DailyMed

See drug prices for KALYDECO

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for KALYDECO

NDA:

207925

Dosage:

GRANULE;ORAL

NDA:

203188

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for KALYDECO

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2
University of Kansas Medical Center	Early Phase 1
University of North Carolina	Early Phase 1

See all KALYDECO clinical trials

Pharmacology for KALYDECO

Drug Class	Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Mechanism of Action	Chloride Channel Activation Potentiators Cytochrome P450 2C9 Inhibitors Cytochrome P450 3A Inhibitors P-Glycoprotein Inhibitors

Paragraph IV (Patent) Challenges for KALYDECO

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
KALYDECO	Oral Granules	ivacaftor	25 mg, 50 mg and 75 mg	207925	1	2022-04-13
KALYDECO	Tablets	ivacaftor	150 mg	203188	1	2020-06-10

US Patents and Regulatory Information for KALYDECO

KALYDECO is protected by thirty-two US patents and seven FDA Regulatory Exclusivities.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-001	Mar 17, 2015		RX	Yes	No			Y	Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015		RX	Yes	Yes
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-002	Mar 17, 2015		RX	Yes	Yes
Vertex Pharms	KALYDECO	ivacaftor	TABLET;ORAL	203188-001	Jan 31, 2012		RX	Yes	Yes			Y	Y
Vertex Pharms Inc	KALYDECO	ivacaftor	GRANULE;ORAL	207925-003	Apr 29, 2019		RX	Yes	No			Y	Y

EU/EMA Drug Approvals for KALYDECO

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Vertex Pharmaceuticals (Ireland) Limited	Kalydeco	ivacaftor	EMEA/H/C/002494 Kalydeco tablets are indicated:As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1).Kalydeco granules are indicated for the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).	Authorised	no	no	no	2012-07-23

International Patents for KALYDECO

When does loss-of-exclusivity occur for KALYDECO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

China

European Patent Office

Hong Kong

Israel

Japan

Mexico

New Zealand

Russian Federation

South Africa

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering KALYDECO around the world.

Country	Patent Number	Title	Estimated Expiration
Lithuania	3219705
Denmark	2502911
Slovenia	1773816
Lithuania	2328618
China	102231990
China	102652128	4 -oxo- iH -quinoline- 3 - carboxamides as modulators of ATP -binding cassette transporters

Supplementary Protection Certificates for KALYDECO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1773816	PA2015028,C1773816	Lithuania		PRODUCT NAME: IVAKAFTORAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA; REGISTRATION NO/DATE: EU/1/12/782/001 - EU/1/12/782/002, 0120723
3170818	2020/035	Ireland		PRODUCT NAME: A COMBINATION OF (A) 3-(6-(1-(2,2-DIFLUOROBENZO(D)(1,3)DIOXOL-5- YL)CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID (LUMACAFTOR) AND (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3- CARBOXAMIDE (IVACAFTOR) OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTRATION NO/DATE: EU/1/15/1059 20151124
1773816	122015000050	Germany		PRODUCT NAME: N-(5-HYDROXY-2,4-DI-TERT-BUTYL-PHENYL)-4-OXO-1H-CHINOLIN-3-CARBONSAEUREAMID ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120723
1773816	300748	Netherlands		PRODUCT NAME: N-(5-HYDROXY-2,4-DI-TERT-BUTYL-FENYL)-4-OXO-1H-CHINOLINE-3-CARBOXAMIDE (INN: IVACAFTOR) OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/12/782/001-002 20120725
1773816	1590036-8	Sweden		PRODUCT NAME: N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3- CARBOXAMIDE OR A PHARMACEUTICALLY ACCEPTABLE SALT THEROF; REG. NO/DATE: EU/1/12/782/001 20120725
2826776	21C1018	France		PRODUCT NAME: TEZACAFTOR ET IVACAFTOR, SOUS TOUTES LES FORMES PROTEGEES PAR LE BREVET DE BASE; REGISTRATION NO/DATE: EU/1/18/1306 20181106

KALYDECO Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory of KALYDECO

Introduction to KALYDECO

KALYDECO, developed by Vertex Pharmaceuticals, is a groundbreaking medication for the treatment of cystic fibrosis (CF), a rare and debilitating genetic disorder. It was first approved by the FDA in 2012 and has since become a cornerstone in the treatment of CF, particularly for patients with specific genetic mutations.

Mechanism and Impact

KALYDECO works by targeting the underlying cause of CF, which is a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The drug, also known as ivacaftor, helps to improve the function of this protein, thereby enhancing lung function and reducing the frequency of pulmonary exacerbations in patients with certain CFTR mutations[4].

Market Dynamics

Rapid Uptake and Expansion

Since its approval, KALYDECO has seen rapid uptake among eligible patients. The drug's initial approval was for patients aged 6 and older with the G551D mutation, but subsequent label expansions have included younger patients and those with other residual function mutations. This expansion has significantly increased the eligible patient pool, driving market growth[3][4].

Dominance in the CF Market

Vertex Pharmaceuticals, the manufacturer of KALYDECO, has maintained a strong dominance in the CF therapeutics market. The company's portfolio, which includes other CF drugs like ORKAMBI and SYMDEKO, as well as the more recent TRIKAFTA, has been instrumental in treating a large percentage of the CF population. KALYDECO, in particular, has been a key contributor to Vertex's revenue growth[1][3].

Global Reimbursement Agreements

Vertex has successfully negotiated reimbursement agreements for KALYDECO in several countries, including France, Spain, Australia, Denmark, Ireland, and Sweden. These agreements have facilitated access to the drug for patients outside the U.S., further boosting its global market presence[1].

Financial Performance

Revenue Growth

KALYDECO has consistently contributed significantly to Vertex's product revenues. In the third quarter of 2017, KALYDECO generated $213.5 million in net product revenues, up from $175.6 million in the same period of 2016. This growth was driven by the approval and uptake among younger patients and those with additional mutations[3].

Annual Revenue Projections

For 2017, Vertex increased its revenue guidance for KALYDECO to $810 to $830 million, reflecting strong demand and expanded label approvals. This trend has continued, with KALYDECO remaining a major revenue driver for Vertex alongside other CF drugs[3].

Comparative Performance

In comparison to other Vertex CF drugs, KALYDECO has maintained a strong market position. While newer drugs like TRIKAFTA have become top-sellers, KALYDECO continues to contribute substantially to Vertex's overall revenue. For example, in the third quarter of 2019, KALYDECO revenues were $236 million, which, although lower than ORKAMBI's $270 million, still represented a significant portion of Vertex's total CF product revenues[2].

Challenges and Restraints

High Cost of Treatment

One of the major challenges facing KALYDECO and other CF treatments is the high cost. The list price for KALYDECO is substantial, which can limit access for some patients. This has led to restrictions and negotiations with governments in various countries to make the drug more affordable[1].

Limited Availability for All Mutations

Despite the progress made, KALYDECO and other current CF treatments do not address all CFTR mutations. This limitation means that a portion of the CF population remains untreated, which is a significant restraint on market growth[1].

Future Outlook

Ongoing Clinical Developments

Vertex continues to invest in clinical development programs aimed at expanding the treatment options for CF patients. New medications and combinations are in various stages of development, which could further enhance the market dynamics and potentially replace or complement KALYDECO in the future[1][3].

Market Projections

The global cystic fibrosis therapeutics market, driven in part by KALYDECO and other Vertex drugs, is anticipated to reach $20.9 billion by 2031, growing at a CAGR of 7.8%. This growth will be fueled by the increasing eligible patient pool, new drug approvals, and ongoing reimbursement agreements[1].

Key Takeaways

• Rapid Uptake and Expansion: KALYDECO has seen rapid uptake since its approval, driven by label expansions and global reimbursement agreements.
• Financial Performance: KALYDECO has been a significant contributor to Vertex's revenue growth, with consistent annual revenue increases.
• Challenges: High treatment costs and limited availability for all CF mutations are key restraints on market growth.
• Future Outlook: Ongoing clinical developments and market projections indicate continued growth in the CF therapeutics market.

FAQs

1. What is KALYDECO used for? KALYDECO is used to treat cystic fibrosis (CF) by improving the function of the CFTR protein in patients with specific genetic mutations.

2. When was KALYDECO approved? KALYDECO was first approved by the FDA in 2012.

3. How much does KALYDECO cost? The cost of KALYDECO is substantial, with prices varying by country and reimbursement agreements.

4. What are the main challenges facing KALYDECO in the market? The main challenges include the high cost of treatment and limited availability for all CFTR mutations.

5. What is the projected market size for CF therapeutics by 2031? The global market for cystic fibrosis therapeutics is anticipated to reach $20.9 billion by 2031, growing at a CAGR of 7.8%[1].

Sources

1. Global Cystic Fibrosis Therapeutics Market $20.9 Billion by 2031 - iHealthcareAnalyst
2. Vertex Reports Full-Year and Fourth-Quarter 2019 Financial Results - Business Wire
3. Vertex Reports Third-Quarter 2017 Financial Results - Vertex Pharmaceuticals
4. Cystic Fibrosis Treatment Market - DelveInsight
5. Vertex Reports Third Quarter 2022 Financial Results - Vertex Pharmaceuticals