ORKAMBI Drug Patent Profile

Which patents cover Orkambi, and when can generic versions of Orkambi launch?

Orkambi is a drug marketed by Vertex Pharms Inc and is included in two NDAs. There are twenty-two patents protecting this drug.

This drug has four hundred and sixty-four patent family members in thirty-six countries.

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

DrugPatentWatch^® Generic Entry Outlook for Orkambi

Orkambi was eligible for patent challenges on July 2, 2019.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be June 11, 2031. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for ORKAMBI

International Patents:	464
US Patents:	22
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	19
Patent Applications:	19
Drug Prices:	Drug price information for ORKAMBI
What excipients (inactive ingredients) are in ORKAMBI?	ORKAMBI excipients list
DailyMed Link:	ORKAMBI at DailyMed

See drug prices for ORKAMBI

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

Constraining patent/regulatory exclusivity:

8,993,600*PED

NDA:

211358

Dosage:

GRANULE;ORAL

Constraining patent/regulatory exclusivity:

8,993,600*PED

NDA:

206038

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 ORKAMBI

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

Sponsor	Phase
Qanatpharma Canada LTD	Phase 1
Children's Hospital Medical Center, Cincinnati	N/A
University of Kansas Medical Center	Early Phase 1

See all ORKAMBI clinical trials

Pharmacology for ORKAMBI

Drug Class	Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Mechanism of Action	Chloride Channel Activation Potentiators Cytochrome P450 2B6 Inducers Cytochrome P450 2C19 Inducers Cytochrome P450 2C8 Inducers Cytochrome P450 2C8 Inhibitors Cytochrome P450 2C9 Inducers Cytochrome P450 2C9 Inhibitors Cytochrome P450 3A Inducers Cytochrome P450 3A Inhibitors P-Glycoprotein Inducers P-Glycoprotein Inhibitors

US Patents and Regulatory Information for ORKAMBI

ORKAMBI is protected by twenty-two US patents and seven FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 8,993,600.

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	ORKAMBI	ivacaftor; lumacaftor	TABLET;ORAL	206038-001	Jul 2, 2015		RX	Yes	Yes	9,931,334*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	9,192,606*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-001	Aug 7, 2018		RX	Yes	No	8,653,103*PED				Y
Vertex Pharms Inc	ORKAMBI	ivacaftor; lumacaftor	GRANULE;ORAL	211358-002	Aug 7, 2018		RX	Yes	Yes

EU/EMA Drug Approvals for ORKAMBI

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Vertex Pharmaceuticals (Ireland) Limited	Orkambi	lumacaftor, ivacaftor	EMEA/H/C/003954 Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene.Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in children aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.	Authorised	no	no	no	2015-11-19

International Patents for ORKAMBI

See the table below for patents covering ORKAMBI around the world.

Country	Patent Number	Title	Estimated Expiration
Australia	2016216569
Brazil	PI0919550	unidades de dosagem de ácido 3-(6-(1-(2,2-difluorobenzo]d][1,3]dioxol-5-il)ciclopropanocarboxamido)-3-metilpiridin-2-il)benzoico
Israel	222314
Serbia	60229	ČVRSTI OBLICI 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOKSOL-5-IL) CIKLOPROPANKARBOKSAMIDO)-3-METILPIRIDIN-2-IL) BENZOJEVE KISELINE (SOLID FORMS OF 3-(6-(1-(2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL) CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL) BENZOIC ACID)

Supplementary Protection Certificates for ORKAMBI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
3170818	301060	Netherlands		PRODUCT NAME: EEN COMBINATIE VAN (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOXOL-5-YL)CYCLOPROPAANCARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOEZUUR EN (B) N-(5-HYDROXY-2,4-DITERT-BUTYL-FENYL)-4-OXO-1H-CHINOLINE-3-CARBOXAMIDE OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/15/1059 20151124
3170818	35/2020	Austria		PRODUCT NAME: EINE KOMBINATION VON (A) 3-(6-(1-(2,2-DIFLUORBENZO(D)(1,3)DIOXOL-5-YL)CYCLOPROPANCARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOESAEURE UND (B) N-(5-HYDROXY-2,4-DITERT-BUTYLPHENYL)-4-OXO-1H-CHINOLIN-3-CARBOXAMID ODER EINEM PHARMAZEUTISCH VERTRAEGLICHEN SALZ DAVON; REGISTRATION NO/DATE: EU/1/15/1059 20151124
1773816	C 2015 027	Romania		PRODUCT NAME: IVACAFTOR SAU O SARE ACCEPTABILA FARMACEUTIC A ACESTUIAN-(5-HIDROXI-2,4-DITERT-BUTIL-FENIL)-4-OXO-1H-CHINOLIN-3-CARBOXAMIDA SAU O SARE ACCEPTABILA FARMACEUTIC AACESTUIA; NATIONAL AUTHORISATION NUMBER: EU/1/12/782/001, EU/1/12/782/002; DATE OF NATIONAL AUTHORISATION: 20120723; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/12/782/001, EU/1/12/782/002; DATE OF FIRST AUTHORISATION IN EEA: 20120723

Market Dynamics and Financial Trajectory of ORKAMBI

Introduction to ORKAMBI

ORKAMBI, developed by Vertex Pharmaceuticals, is a groundbreaking medication designed to treat the underlying cause of cystic fibrosis (CF), a genetic disorder that affects the lungs and digestive system. The drug, a combination of lumacaftor and ivacaftor, has significantly transformed the CF therapeutic landscape since its approval.

Approval and Expansion of Indications

ORKAMBI was first approved by the FDA in January 2012 for patients with the G551D mutation who are at least 6 years old. This initial approval marked the beginning of a new era in CF treatment, targeting the root cause of the disease rather than just its symptoms.

• 2014: ORKAMBI received approval for patients with the R117H and other gating mutations, further expanding its patient pool[1].
• 2015: The drug was approved for patients with the F508Del mutation who are at least 12 years old, and later in the year, it was approved for patients who are at least 6 years old[1].
• 2016: Health Canada granted market authorization for ORKAMBI to include use in children ages 2 through 5 years with CF who have two copies of the F508Del mutation[5].

Clinical Impact and Patient Outcomes

The introduction of ORKAMBI has led to significant improvements in patient outcomes. Clinical trials have shown that the drug increases lung function, as measured by FEV1 (forced expiratory volume in one second), and reduces pulmonary exacerbations.

• FEV1 Improvement: ORKAMBI has been shown to improve FEV1 by more than 2.8%, a significant clinical benefit for CF patients[3].
• Early Screening and Treatment: Early screening and the availability of ORKAMBI have contributed to an increase in the survival of CF patients and improved the overall pace of market size growth[1].

Financial Performance

The financial trajectory of ORKAMBI has been robust, driven by its clinical efficacy and expanding patient base.

• 2016: Net product revenues from ORKAMBI were $336.2 million in the third quarter, a significant increase from $234.0 million in the third quarter of 2016. This growth was driven by the continued uptake in children with CF ages 6 to 11 in the U.S. and additional revenues from European countries where ORKAMBI is reimbursed[4].
• 2017: Vertex increased its total 2017 CF product revenue guidance to $2.10 to $2.15 billion, with ORKAMBI revenues expected to be between $1.29 to $1.32 billion. This updated guidance reflected strong demand for ORKAMBI among people with CF ages 6 and older in the U.S. and potential revenues from countries where the drug is reimbursed[4].
• 2020: The overall financial performance of Vertex Pharmaceuticals, largely driven by ORKAMBI and other CF drugs like Symdeko and Trikafta, saw GAAP and Non-GAAP product revenues increase by 49% and 55%, respectively, compared to 2019. This growth was primarily driven by the uptake of Trikafta, but ORKAMBI continued to contribute significantly to the company's revenues[2].

Market Dynamics and Competition

The CF therapeutic market has become increasingly competitive, but ORKAMBI remains a key player.

• Successor Drugs: Vertex has introduced successor drugs like Symdeko (tezacaftor/ivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor), which have shown even greater efficacy. Symdeko, for example, has demonstrated a 4% increase in FEV1, outperforming ORKAMBI's 2.8% improvement[3].
• Global Expansion: Despite the emergence of new treatments, ORKAMBI continues to be approved and reimbursed in various countries, ensuring its continued relevance in the global CF market[5].

Challenges and Controversies

While ORKAMBI has been a game-changer, it has not been without challenges.

• Pricing Controversies: The drug has faced pricing controversies, particularly in the UK, where it was not pursued for placement on the National Health Service (NHS) due to cost concerns[3].
• CEO Transition: The transition from CEO Jeffrey Leiden to Reshma Kewalramani raised concerns about potential stagnant growth, although the company has maintained its strong pipeline and financial performance[3].

Future Outlook

The future outlook for ORKAMBI and Vertex Pharmaceuticals remains positive, despite the emergence of new treatments.

• Pipeline Development: Vertex continues to develop new candidates, including VX-445, and is expanding into other disease areas such as alpha-1 antitrypsin, sickle cell disease, and beta thalassemia[3].
• Market Growth: The CF market is expected to continue growing, driven by early screening, new therapies, and an increasing eligible patient pool[1].

Key Takeaways

• ORKAMBI has significantly transformed the CF therapeutic landscape with its approvals and expansions.
• The drug has shown substantial clinical benefits, improving lung function and reducing pulmonary exacerbations.
• Financially, ORKAMBI has been a major contributor to Vertex Pharmaceuticals' revenue growth.
• Despite competition from successor drugs, ORKAMBI remains a crucial part of the CF treatment arsenal.
• Pricing controversies and CEO transitions have posed challenges, but the company's pipeline and financial performance remain strong.

FAQs

What is ORKAMBI used for?

ORKAMBI is used to treat the underlying cause of cystic fibrosis (CF) in patients with specific mutations, particularly the F508Del mutation.

When was ORKAMBI first approved?

ORKAMBI was first approved by the FDA in January 2012 for patients with the G551D mutation who are at least 6 years old.

How has ORKAMBI impacted patient outcomes?

ORKAMBI has improved lung function, as measured by FEV1, and reduced pulmonary exacerbations, leading to better survival rates and quality of life for CF patients.

What are the financial highlights of ORKAMBI?

ORKAMBI has contributed significantly to Vertex Pharmaceuticals' revenue growth, with net product revenues increasing substantially since its approval. In 2017, ORKAMBI revenues were expected to be between $1.29 to $1.32 billion.

Are there any successor drugs to ORKAMBI?

Yes, Vertex has introduced successor drugs like Symdeko and Trikafta, which have shown greater efficacy and are now driving the company's revenue growth.

Sources

1. DelveInsight: Cystic Fibrosis Treatment Market.
2. Vertex Pharmaceuticals: Full-Year and Fourth-Quarter 2020 Financial Results.
3. FiercePharma: Vertex touts Symdeko success in face of CEO turnover, Orkambi stumble.
4. Vertex Pharmaceuticals: Third-Quarter 2017 Financial Results.
5. Vertex Pharmaceuticals: Health Canada Grants Market Authorization for ORKAMBI.