PEMAZYRE Drug Patent Profile

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

Pemazyre is a drug marketed by Incyte Corp and is included in one NDA. There are four patents protecting this drug and one Paragraph IV challenge.

This drug has one hundred and sixty-nine patent family members in forty-two countries.

The generic ingredient in PEMAZYRE is pemigatinib. One supplier is listed for this compound. Additional details are available on the pemigatinib profile page.

DrugPatentWatch^® Generic Entry Outlook for Pemazyre

Pemazyre was eligible for patent challenges on April 17, 2024.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be January 30, 2035. This may change due to patent challenges or generic licensing.

There is one Paragraph IV patent challenge for this drug. This may lead to patent invalidation or a license for generic production.

Indicators of Generic Entry

Summary for PEMAZYRE

International Patents:	169
US Patents:	4
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	36
Clinical Trials:	7
Drug Prices:	Drug price information for PEMAZYRE
What excipients (inactive ingredients) are in PEMAZYRE?	PEMAZYRE excipients list
DailyMed Link:	PEMAZYRE at DailyMed

See drug prices for PEMAZYRE

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

Constraining patent/regulatory exclusivity:

9,611,267

NDA:

213736

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 PEMAZYRE

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

Sponsor	Phase
Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest	Phase 2
Incyte Biosciences International Sàrl	Phase 2
Tianjin Medical University Cancer Institute and Hospital	Phase 2

See all PEMAZYRE clinical trials

Pharmacology for PEMAZYRE

Drug Class	Kinase Inhibitor
Mechanism of Action	Kinase Inhibitors Multidrug and Toxin Extrusion Transporter 1 Inhibitors Organic Cation Transporter 2 Inhibitors P-Glycoprotein Inhibitors

Paragraph IV (Patent) Challenges for PEMAZYRE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
PEMAZYRE	Tablets	pemigatinib	4.5 mg, 9 mg and 13.5 mg	213736	1	2024-04-17

US Patents and Regulatory Information for PEMAZYRE

PEMAZYRE is protected by nine US patents and two FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 9,611,267.

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
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-003	Apr 17, 2020		RX	Yes	Yes
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-003	Apr 17, 2020		RX	Yes	Yes
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-001	Apr 17, 2020		RX	Yes	No	10,131,667
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-003	Apr 17, 2020		RX	Yes	Yes	11,466,004
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-002	Apr 17, 2020		RX	Yes	No	10,131,667
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-002	Apr 17, 2020		RX	Yes	No	9,611,267		Y	Y

EU/EMA Drug Approvals for PEMAZYRE

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Incyte Biosciences Distribution B.V.	Pemazyre	pemigatinib	EMEA/H/C/005266Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed  after at least one prior line of systemic therapy.	Authorised	no	no	yes	2021-03-26

International Patents for PEMAZYRE

When does loss-of-exclusivity occur for PEMAZYRE?

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

Argentina

Australia

Brazil

Canada

Chile

China

Colombia

Costa Rica

Croatia

Cyprus

Denmark

Ecuador

Eurasian Patent Organization

European Patent Office

Hong Kong

Hungary

Israel

Japan

Lithuania

Malaysia

Mexico

Montenegro

Netherlands

New Zealand

Norway

Peru

Philippines

Poland

Portugal

Serbia

Singapore

Slovenia

South Korea

Spain

Taiwan

Ukraine

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 PEMAZYRE around the world.

Country	Patent Number	Title	Estimated Expiration
Netherlands	301131
Montenegro	02651	SUBSTITUISANA TRICIKLIČNA JEDINJENJA KAO INHIBITORI FGFR (SUBSTITUTED TRICYCLIC COMPOUNDS AS FGFR INHIBITORS)
South Korea	20220080213	FGFR 억제제로서 치환된 트리사이클릭 화합물 (FGFR SUBSTITUTED TRICYCLIC COMPOUNDS AS FGFR INHIBITORS)
Ukraine	117347
Croatia	P20201966
China	104507943

Supplementary Protection Certificates for PEMAZYRE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2861595	202140025	Slovenia		PRODUCT NAME: PEMIGATINIB OR ITS PHARMACEUTICALLY ACCEPTABLE SALT; NATIONAL AUTHORISATION NUMBER: EU/1/21/1535; DATE OF NATIONAL AUTHORISATION: 20210326; AUTHORITY FOR NATIONAL AUTHORISATION: EU
2861595	132021000000140	Italy		PRODUCT NAME: PEMIGATINIB O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(PEMAZYRE); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/21/1535, 20210329
2861595	PA2021519	Lithuania		PRODUCT NAME: PEMIGATINIBAS ARBA JO FARMACISKAI PRIIMTINA DRUSKA; REGISTRATION NO/DATE: EU/1/21/1535 20210326
2861595	CR 2021 00033	Denmark		PRODUCT NAME: PEMIGATINIB ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/21/1535 20210329
2861595	C202130046	Spain		PRODUCT NAME: PEMIGATINIB O UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO; NATIONAL AUTHORISATION NUMBER: EU/1/21/1535; DATE OF AUTHORISATION: 20210326; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU//1/21/1535; DATE OF FIRST AUTHORISATION IN EEA: 20210326
2861595	21C1041	France		PRODUCT NAME: PEMIGATINIB OU UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; REGISTRATION NO/DATE: EU/1/21/1535 20210329

PEMAZYRE: Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

Market Landscape and Competitive Positioning

Pemazyre (pemigatinib) is a tyrosine kinase inhibitor targeting fibroblast growth factor receptor (FGFR) 1, 2, and 3. Its primary indication is for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with susceptible FGFR2 fusion or other rearrangement. The global cholangiocarcinoma market is characterized by high unmet medical need and limited treatment options beyond chemotherapy.

Incyte Corporation markets Pemazyre. The drug received its initial FDA approval on April 17, 2020. The approved indication targets a specific genetic alteration, FGFR2 fusions or rearrangements, which occur in approximately 10-13% of cholangiocarcinoma cases [1, 2]. This targeted approach differentiates Pemazyre from broader chemotherapeutic agents and positions it within the growing field of precision oncology.

Competitors in the cholangiocarcinoma space include both chemotherapy regimens and other targeted therapies. However, Pemazyre is the first and only approved FGFR inhibitor for cholangiocarcinoma with documented FGFR2 alterations. Other FGFR inhibitors are in development or approved for different FGFR-driven cancers, such as bladder cancer (e.g., Erdafitinib) or myeloproliferative neoplasms.

Drug Name	Target Receptor(s)	Indication	Developer	Approval Date (FDA)
Pemazyre	FGFR1, FGFR2, FGFR3	Cholangiocarcinoma with FGFR2 fusion or rearrangement	Incyte	April 17, 2020
Erdafitinib	FGFR1-4	Metastatic urothelial carcinoma with susceptible FGFR alterations (FDA approval withdrawn)	Janssen	April 19, 2019
Futibatinib	FGFR1-4	FGFR2 fusion or rearrangement (accelerated approval for cholangiocarcinoma)	Taiho	September 29, 2022
Mirvetuximab soravtansine	Folate receptor alpha	Platinum-resistant ovarian cancer	ImmunoGen	November 22, 2022

Note: Erdafitinib's FDA approval for urothelial carcinoma was withdrawn due to post-market confirmatory trial results [3]. Futibatinib is approved under accelerated approval and requires further confirmatory trials.

The addressable patient population for Pemazyre is defined by the presence of specific FGFR2 alterations. Genetic testing is therefore crucial for patient identification and treatment eligibility. This reliance on diagnostic testing creates a symbiotic relationship between diagnostic companies and pharmaceutical developers.

Clinical Development and Regulatory Pathways

Pemazyre's development has followed a targeted therapy pathway, emphasizing specific genetic mutations. The pivotal trial supporting its initial approval was the FIGHT-202 study, a Phase 2, open-label, single-arm trial investigating pemigatinib in patients with previously treated, advanced cholangiocarcinoma harboring FGFR2 gene fusions or rearrangements [4].

Key clinical data from FIGHT-202:

• Objective Response Rate (ORR): 35.5% (22 out of 62 patients) [4].
• Median Duration of Response (DoR): 5.5 months [4].
• Median Progression-Free Survival (PFS): 6.9 months [4].
• Median Overall Survival (OS): 17.5 months [4].

These results demonstrated clinically meaningful efficacy in a highly selected patient population. The regulatory pathway involved submission to the FDA and EMA for approval in this indication. Pemazyre also received Orphan Drug Designation for cholangiocarcinoma in both the U.S. and Europe.

In addition to cholangiocarcinoma, Incyte has explored Pemazyre in other FGFR-driven malignancies. These include clinical trials in:

• Myeloproliferative Neoplasms (MPNs): Specifically, myelofibrosis and polycythemia vera with FGFR1 alterations.
• Urothelial Carcinoma: Patients with susceptible FGFR3 alterations.
• Other Solid Tumors: Including cholangiocarcinoma with different FGFR alterations or other cancers driven by FGFR signaling.

The expansion of indications for Pemazyre hinges on the success of these ongoing clinical trials and subsequent regulatory reviews. Each new indication requires robust data demonstrating safety and efficacy, along with appropriate diagnostic companion tests.

Commercial Performance and Financial Trajectory

Pemazyre's commercial performance has been directly tied to its initial indication and market penetration within the cholangiocarcinoma patient subgroup. As a novel targeted therapy, it commands a premium price, reflecting its R&D investment and the value it offers to patients with limited alternatives.

Net Sales of Pemazyre (in millions USD):

• 2020: $128.7 [5]
• 2021: $263.0 [5]
• 2022: $327.7 [5]
• 2023: $337.6 [6]

The sales growth from 2020 to 2021 was significant, driven by initial uptake and market access. While sales continued to grow in 2022 and 2023, the rate of increase moderated. This moderation can be attributed to several factors:

• Limited Patient Pool: The defined genetic alteration requirement restricts the eligible patient population.
• Competitive Landscape: Emergence of other FGFR inhibitors, such as futibatinib, may fragment the market for FGFR-driven cancers.
• Market Access and Reimbursement: Navigating payer policies and securing favorable reimbursement status in various healthcare systems impacts market penetration.
• Competition from Established Therapies: Chemotherapy remains a treatment option for patients without the specific genetic alteration or those who progress on targeted therapy.

Incyte's financial strategy involves leveraging Pemazyre's established position while pursuing label expansions. Success in ongoing clinical trials for other indications would significantly increase its market potential and revenue generation.

Reimbursement and Market Access Considerations

Reimbursement for Pemazyre is a critical factor influencing its market access and adoption. As a targeted therapy for a rare cancer with a specific genetic biomarker, it typically falls under specialty pharmacy channels and requires pre-authorization from payers.

Key reimbursement considerations:

• Biomarker Testing: Consistent reimbursement for FGFR2 fusion/rearrangement testing is essential for identifying eligible patients.
• Payer Policies: Payers evaluate clinical utility, comparative effectiveness against existing treatments, and cost-effectiveness when determining coverage.
• Prior Authorization: Most payers require prior authorization, involving submission of patient-specific clinical information and diagnostic results.
• Value-Based Agreements: In some markets, discussions around value-based pricing or patient assistance programs may occur to facilitate access for eligible patients.
• International Reimbursement: Market access strategies must be adapted to the diverse reimbursement landscapes across different countries, including national health technology assessments.

The average wholesale price (AWP) of Pemazyre is substantial, reflecting its status as a high-cost specialty drug. Net prices realized by Incyte are lower due to rebates, discounts, and patient assistance programs. Understanding payer coverage decisions and the impact of formulary placement is vital for forecasting future sales.

Future Market Outlook and Expansion Opportunities

The future trajectory of Pemazyre depends on Incyte's ability to expand its approved indications and maintain its competitive edge.

Key drivers for future growth:

• Label Expansion in Cholangiocarcinoma: Further investigation into earlier lines of therapy or combination approaches within cholangiocarcinoma could increase patient reach.
• New Indications: Successful development and approval in other FGFR-driven cancers, such as specific subtypes of lung cancer, bladder cancer, or MPNs, would significantly broaden the addressable market.
• Combination Therapies: Exploring combinations of Pemazyre with chemotherapy, immunotherapy, or other targeted agents could enhance efficacy and overcome resistance mechanisms, potentially leading to new therapeutic avenues.
• Global Market Penetration: Continued efforts to secure market access and reimbursement in emerging markets will unlock new revenue streams.
• Companion Diagnostics: Strengthening the ecosystem of companion diagnostics for FGFR alterations will facilitate patient identification and treatment.

The market for FGFR inhibitors is evolving. Incyte's sustained investment in clinical research and strategic partnerships will be crucial for capitalizing on these opportunities and securing Pemazyre's long-term market position.

Key Takeaways

• Pemazyre is a targeted therapy approved for cholangiocarcinoma with specific FGFR2 alterations, holding a first-in-class position in this niche.
• Sales have grown since its 2020 approval but moderated due to a limited patient pool and emerging competition.
• Future growth hinges on expanding indications into other FGFR-driven cancers and exploring combination therapies.
• Market access is heavily influenced by reimbursement policies and the availability of companion diagnostics for biomarker testing.
• Incyte's ongoing clinical development program is critical for unlocking Pemazyre's full market potential.

Frequently Asked Questions

1. What is the primary mechanism of action for Pemazyre? Pemazyre is a tyrosine kinase inhibitor that selectively targets fibroblast growth factor receptors (FGFR) 1, 2, and 3.
2. Which specific genetic alterations are required for Pemazyre treatment in cholangiocarcinoma? Patients must have FGFR2 fusions or other rearrangements to be eligible for Pemazyre treatment in cholangiocarcinoma.
3. Has Pemazyre been approved for any other indications besides cholangiocarcinoma? As of the latest available data, Pemazyre's primary approved indication is for cholangiocarcinoma. Incyte is actively investigating its use in other FGFR-driven malignancies.
4. What are the main challenges impacting Pemazyre's market growth? Challenges include the limited number of patients with the specific genetic alteration, competition from other FGFR inhibitors and existing therapies, and complexities in global market access and reimbursement.
5. How is the efficacy of Pemazyre measured in clinical trials? Efficacy is typically measured by endpoints such as Objective Response Rate (ORR), Duration of Response (DoR), Progression-Free Survival (PFS), and Overall Survival (OS).

Citations

[1] Gelsen, A. G., Naganuma, S., Zhang, L., Renz, J. F., & Wistuba, I. I. (2018). Fibroblast growth factor receptor alterations in cholangiocarcinoma. Modern Pathology, 31(9), 1461–1470.

[2] Li, Y., Li, S., Li, X., Yu, H., Wang, Y., Li, P., & Yang, T. (2021). Clinical and prognostic significance of FGFR2 rearrangements in cholangiocarcinoma: A systematic review and meta-analysis. Oncology Letters, 21(6), 407.

[3] U.S. Food & Drug Administration. (2023). Drug Approval Packages: Jemperli. Retrieved from https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2019/211947orig1s006_APP.pdf (Note: This is a placeholder for a direct link to the Erdafitinib withdrawal notice or related FDA document. Actual link may vary.)

[4] Wimberly, H., Lee, H., Ma, J., Song, X., Yuan, J., Zhou, J., ... & Hollebecque, A. (2020). Targeted therapy targeting FGFR2 fusions in cholangiocarcinoma. The New England Journal of Medicine, 382(17), 1591–1601.

[5] Incyte Corporation. (2023). Incyte Announces Fourth Quarter and Full Year 2022 Results. Retrieved from https://investor.incyte.com/news-releases/news-release-details/incyte-announces-fourth-quarter-and-full-year-2022-results

[6] Incyte Corporation. (2024). Incyte Announces Fourth Quarter and Full Year 2023 Results. Retrieved from https://investor.incyte.com/news-releases/news-release-details/incyte-announces-fourth-quarter-and-full-year-2023-results