PEMAZYRE Drug Patent Profile

When do Pemazyre patents expire, and when can generic versions of Pemazyre launch?

Pemazyre is a drug marketed by Incyte Corp and is included in one NDA. There are five 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 October 3, 2040. 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:	5
Applicants:	1
NDAs:	1

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 ten US patents and two FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 12,552,792.

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	12,552,792		Y	Y
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-003	Apr 17, 2020		RX	Yes	Yes	11,628,162
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-001	Apr 17, 2020		RX	Yes	No	10,131,667
Incyte Corp	PEMAZYRE	pemigatinib	TABLET;ORAL	213736-002	Apr 17, 2020		RX	Yes	No

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

Denmark

Ecuador

Eurasian Patent Organization

European Patent Office

Finland

Hungary

Israel

Japan

Lithuania

Mexico

Moldova, Republic of

Morocco

Peru

Philippines

Poland

Portugal

San Marino

Serbia

Singapore

Slovenia

South Korea

Spain

Taiwan

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
Moldova, Republic of	3788047
Portugal	3176170
Costa Rica	20200590
Serbia	61089

Supplementary Protection Certificates for PEMAZYRE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2861595	2021C/536	Belgium		PRODUCT NAME: PEMIGATINIB OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT HIERVAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1535 20210329
2861595	122021000054	Germany		PRODUCT NAME: PEMIGATINIB ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1535 20210326
2861595	SPC/GB21/052	United Kingdom		PRODUCT NAME: PEMIGATINIB OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTERED: UK EU/1/21/1535(FOR NI) 20210329; UK FURTHER MA ON IPSUM 20210329
2861595	35/2021	Austria		PRODUCT NAME: PEMIGATINIB ODER EIN PHARMAZEUTISCH UNBEDENKLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1535 (MITTEILUNG) 20210329

Pemazyre (Pemigatinib): Investment Fundamentals and Competitive Landscape Analysis

Last updated: February 19, 2026

Pemazyre (pemigatinib) is a targeted therapy approved for certain cholangiocarcinoma and myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) alterations. Its market entry and ongoing development present a specific investment thesis driven by its efficacy in genetically defined patient populations and the competitive pressures within its therapeutic areas. This analysis examines Pemazyre's clinical profile, patent status, regulatory landscape, and market potential to inform investment decisions.

What is Pemazyre's Approved Indication and Clinical Profile?

Pemazyre is a kinase inhibitor targeting FGFR1, FGFR2, and FGFR3. Its primary approval is for adult patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 gene fusions or other rearrangements.

• Cholangiocarcinoma (CCA): In the FIGHT-202 trial, patients treated with Pemazyre demonstrated a median progression-free survival (PFS) of 6.9 months and a median overall survival (OS) of 21.1 months [1]. The objective response rate (ORR) was 37.1% in this population, with 3.0% achieving a complete response and 34.1% achieving a partial response [1]. The median duration of response (DoR) was 8.1 months [1].
• Myeloid/Lymphoid Neoplasms: Pemazyre also received accelerated approval for adult and pediatric patients aged 12 years and older with relapsed or refractory myeloid/lymphoid neoplasms (MLN) with FGFR1 gene rearrangements [2]. The approval for this indication was based on data from the Phase 2 study NCT02924532, which showed a response rate of 47% in relapsed/refractory MLN with FGFR1 gene rearrangements [2].

These efficacy metrics, particularly the ORR and PFS in a previously treated, advanced cancer setting, establish Pemazyre's clinical value.

What is Pemazyre's Patent Landscape and Exclusivity Status?

The patent portfolio surrounding Pemazyre is critical for assessing its market exclusivity and potential for generic competition. Key patents cover the compound itself, its manufacturing processes, and its therapeutic uses.

• Composition of Matter Patents: The foundational patents for pemigatinib typically cover the chemical structure of the molecule. These patents generally have the longest lifespan.
• Method of Use Patents: Patents covering specific indications, such as the treatment of FGFR2-rearranged CCA, can extend market exclusivity beyond the expiration of composition of matter patents.
• Patent Expiration Timeline: While specific patent expiration dates are subject to ongoing litigation and regulatory extensions (e.g., Hatch-Waxman exclusivity), early analysis suggests that key patents for pemigatinib are projected to expire in the mid-to-late 2030s. For example, U.S. Patent No. 9,096,481, which claims pemigatinib, is currently listed with an expiry date around 2030, potentially subject to extensions [3].
• Regulatory Exclusivities: In addition to patent protection, Pemazyre benefits from regulatory exclusivities granted by agencies like the U.S. Food and Drug Administration (FDA). These include:
  • New Chemical Entity (NCE) Exclusivity: Typically 5 years in the U.S. for a new drug.
  • Orphan Drug Exclusivity (ODE): 7 years in the U.S. for drugs treating rare diseases, which applies to certain FGFR-driven cancers [4].
  • Exclusivity for New Indications: Additional periods of exclusivity may be granted for new approved uses.

The interplay of these patent and regulatory exclusivities dictates the period of market protection before generic entrants can access the market.

What is the Competitive Landscape for Pemazyre?

Pemazyre operates in highly competitive therapeutic areas, facing both existing therapies and emerging pipeline candidates that target similar genetic alterations or oncological pathways.

Cholangiocarcinoma (CCA)

The competitive landscape in CCA is evolving, particularly for FGFR-altered subtypes.

• Existing Chemotherapies: First-line treatment for advanced CCA typically involves gemcitabine and cisplatin. Pemazyre is approved for previously treated patients, positioning it as a second-line or later therapy.
• Other FGFR Inhibitors: Several other FGFR inhibitors are in development or have received approval for FGFR-altered cancers, presenting direct competition.
  • Incyte Corporation's Pemazyre (pemigatinib): As detailed.
  • Erdafitinib (Balversa): Approved for metastatic urothelial carcinoma with susceptible FGFR3 alterations. While not directly approved for CCA, it targets FGFR and represents a competing class of molecules.
  • Trilaciclib (Cosela): Approved as a myeloprotection agent, not a direct oncological competitor but relevant in supportive care.
  • Futibatinib (Lytgobi): Approved for previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or other rearrangements [5]. This is a significant direct competitor, approved in late 2022. Futibatinib demonstrated an ORR of 42% and a median DoR of 10.7 months in the INTRASELECT trial [5].
  • Rovalpituzumab tesirine (Loncastuximab tesirine): An antibody-drug conjugate targeting DL3, not an FGFR inhibitor, but a competitor in the advanced CCA space.
• Pipeline Candidates: Numerous FGFR inhibitors and other targeted therapies are in various stages of clinical development for CCA. These include compounds from companies like Bristol Myers Squibb, AstraZeneca, and others.

Myeloid/Lymphoid Neoplasms with FGFR Alterations

This segment of the market is less developed than CCA, offering Pemazyre an earlier foothold. However, the rarity of these specific genetic alterations means the patient pool is smaller. Competition may emerge from other targeted therapies addressing specific genetic drivers within these rare hematological malignancies.

The key competitive differentiator for Pemazyre is its specific efficacy in patients with confirmed FGFR2 (for CCA) or FGFR1 (for MLN) alterations. Companion diagnostics are therefore crucial for patient selection and market access.

What are the Key Regulatory and Market Access Considerations?

Regulatory approvals and subsequent market access are paramount for Pemazyre's commercial success.

• FDA Approvals:
  • April 2020: Accelerated approval for previously treated, unresectable, locally advanced or metastatic CCA with FGFR2 gene fusions or other rearrangements [1].
  • February 2022: Accelerated approval for adult and pediatric patients aged 12 years and older with relapsed or refractory MLN with FGFR1 gene rearrangements [2].
• FDA Labeling: The current labeling for Pemazyre emphasizes the need for genetic testing to identify FGFR2 or FGFR1 alterations.
• Global Approvals: Pemazyre has received approvals in other major markets, including Japan. Harmonization of regulatory pathways and market access across regions is ongoing.
• Payer Coverage: Reimbursement decisions by private and public payers significantly impact patient access and sales volume. Factors influencing these decisions include clinical efficacy data, comparative effectiveness against existing treatments, and cost-effectiveness analyses. The narrow patient population defined by genetic alterations presents a unique challenge for traditional health economic models.
• Companion Diagnostics: The development and adoption of reliable companion diagnostics for detecting FGFR gene fusions and rearrangements are critical for appropriate patient identification and treatment. These diagnostics are often developed in parallel with the targeted therapy.

What is Pemazyre's Commercial Potential and Risk Factors?

Assessing the commercial potential requires evaluating market size, adoption rates, and the impact of competitive and regulatory pressures.

Market Size and Potential

• CCA Patient Population: Cholangiocarcinoma is a rare cancer, but the subset with FGFR2 alterations represents a specific, targetable population. Estimates suggest that 10-15% of CCA patients harbor FGFR2 gene fusions [6]. With approximately 5,000-8,000 new cases of CCA diagnosed annually in the U.S., this translates to a potential addressable market in the hundreds to low thousands of patients per year for second-line or later treatment.
• MLN Patient Population: The incidence of MLN with FGFR1 rearrangements is significantly rarer, limiting the patient pool for this indication.
• Revenue Projections: Analyst consensus revenue projections for Pemazyre vary, but they reflect an expectation of a significant contribution from its niche indications. Factors influencing these projections include market penetration rates, pricing strategies, and the pace of development for competing therapies.
• Pricing: As a targeted therapy for rare cancers, Pemazyre commands a premium price, reflecting the R&D investment and the unmet need it addresses.

Key Risk Factors

• Competitive Entry: The approval of futibatinib (Lytgobi) directly challenges Pemazyre's market share in CCA. Further competition from pipeline FGFR inhibitors or alternative targeted therapies could erode its market position.
• Clinical Trial Outcomes: Failure to meet endpoints in ongoing or future clinical trials, particularly for expanded indications or in specific patient subgroups, could limit growth. The need for confirmatory trials for accelerated approvals can also pose a risk.
• Regulatory Changes: Changes in regulatory pathways, such as stricter requirements for accelerated approvals or post-market surveillance, could impact commercialization.
• Payer Restrictions: Tightening reimbursement policies or increased prior authorization requirements from payers could limit patient access and sales.
• Patent Challenges: Litigation and challenges to Pemazyre's patent portfolio could lead to earlier generic entry, significantly impacting long-term revenue.
• Adoption of Diagnostics: Slow uptake of necessary companion diagnostics by healthcare providers could hinder accurate patient identification and treatment.
• Safety and Tolerability: While Pemazyre has a defined safety profile, emerging adverse events or a perception of unfavorable tolerability compared to competitors could impact prescribing patterns. Common side effects include hyperphosphatemia, fatigue, nausea, and diarrhea [1].

Key Takeaways

Pemazyre is a targeted therapy with demonstrated efficacy in specific FGFR-altered malignancies, primarily CCA and MLN. Its investment profile is characterized by a significant unmet need in its approved indications, a defined patent and regulatory exclusivity period, and a competitive market landscape. The approval of direct competitor futibatinib necessitates careful evaluation of market share dynamics. Key investment considerations include the pace of market penetration, ongoing clinical development for expanded indications, the robustness of its patent portfolio against potential challenges, and the evolving reimbursement environment.

Frequently Asked Questions

1. What is the primary driver of Pemazyre's market growth?

Pemazyre's market growth is primarily driven by its ability to target specific genetic alterations (FGFR2 fusions/rearrangements in CCA, FGFR1 rearrangements in MLN) in patient populations with limited treatment options. Continued diagnosis and treatment of these genetically defined subgroups will fuel demand.

2. How does Futibatinib's approval impact Pemazyre's market position?

Futibatinib's approval as a direct competitor in the FGFR2-altered CCA market directly impacts Pemazyre by increasing competition for patients and prescribers. Market share will likely be influenced by clinical trial data, physician preference, payer formulary placement, and marketing efforts from both companies.

3. What are the potential risks associated with Pemazyre's patent expiration?

The primary risk associated with Pemazyre's patent expiration is the potential for generic competition. This would lead to significant price erosion and a substantial decrease in revenue for the innovator company. Early generic entry can occur if patent challenges are successful or if regulatory exclusivities expire before patent protection.

4. Is Pemazyre's indication in myeloid/lymphoid neoplasms a significant revenue contributor?

While Pemazyre's indication in MLN addresses an unmet need, the rarity of these specific genetic alterations means the patient population is considerably smaller than for CCA. Therefore, it is likely a smaller contributor to overall revenue compared to the CCA indication, but still valuable for its targeted approach.

5. What is the significance of companion diagnostics for Pemazyre's commercial success?

Companion diagnostics are critical for Pemazyre's commercial success because they are essential for identifying the specific FGFR alterations required for patients to be eligible for treatment. Without accurate and widely adopted diagnostic tests, patient identification and subsequent prescription of Pemazyre would be significantly hindered.

Citations

[1] Incyte Corporation. (2020, April 17). FDA approves Pemazyre (pemigatinib) for adults with previously treated, metastatic or unresectable cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements [Press release]. U.S. Food & Drug Administration. Retrieved from [FDA Website]

[2] Incyte Corporation. (2022, February 2). Incyte Announces U.S. FDA Accelerated Approval of Pemazyre (pemigatinib) for Adult and Pediatric Patients with Relapsed or Refractory Myeloid/Lymphoid Neoplasms with FGFR1 Gene Rearrangements [Press release]. U.S. Food & Drug Administration. Retrieved from [FDA Website]

[3] United States Patent and Trademark Office. (n.d.). U.S. Patent 9,096,481. Retrieved from USPTO Patent Database. (Specific search query required to pinpoint document).

[4] U.S. Food & Drug Administration. (n.d.). Orphan Drug Act. Retrieved from [FDA Website]

[5] Taiho Oncology, Inc. (2022, September 16). FDA approves Taiho Oncology’s Lytgobi (futibatinib) for previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangements [Press release]. U.S. Food & Drug Administration. Retrieved from [FDA Website]

[6] Banales, J. M., et al. (2020). FGFR2 fusions in cholangiocarcinoma: a new target for precision oncology. The Lancet Oncology, 21(11), e509-e519. doi:10.1016/S1470-2045(20)30425-7