PYRUKYND Drug Patent Profile

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

Pyrukynd is a drug marketed by Agios Pharms Inc and is included in one NDA. There are nine patents protecting this drug and one Paragraph IV challenge.

This drug has one hundred and ninety-three patent family members in forty-six countries.

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

DrugPatentWatch^® Generic Entry Outlook for Pyrukynd

Pyrukynd was eligible for patent challenges on February 17, 2026.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be April 11, 2035. 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.

Indicators of Generic Entry

Summary for PYRUKYND

International Patents:	193
US Patents:	9
Applicants:	1
NDAs:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for PYRUKYND

Paragraph IV (Patent) Challenges for PYRUKYND

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
PYRUKYND	Tablets	mitapivat sulfate	5 mg, 20 mg and 50 mg	216196	1	2026-02-17

US Patents and Regulatory Information for PYRUKYND

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

This potential generic entry date is based on patent RE49582.

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
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-003	Feb 17, 2022		RX	Yes	Yes
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-003	Feb 17, 2022		RX	Yes	Yes	10,632,114
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-003	Feb 17, 2022		RX	Yes	Yes
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-003	Feb 17, 2022		RX	Yes	Yes	11,234,976
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-001	Feb 17, 2022		RX	Yes	No	11,878,049
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-002	Feb 17, 2022		RX	Yes	No	11,234,976
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-003	Feb 17, 2022		RX	Yes	Yes	11,793,806

International Patents for PYRUKYND

When does loss-of-exclusivity occur for PYRUKYND?

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

European Patent Office

Finland

France

Hungary

India

Israel

Japan

Lithuania

Malaysia

Mexico

New Zealand

Nicaragua

Norway

Peru

Poland

Portugal

Russian Federation

Singapore

Slovenia

South Africa

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 PYRUKYND around the world.

Country	Patent Number	Title	Estimated Expiration
Australia	2012250688
Australia	2017221860
Australia	2020200700
Brazil	112013028422
Canada	2834602
Canada	3088328
China	103764147

Supplementary Protection Certificates for PYRUKYND

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2448582	202340010	Slovenia		PRODUCT NAME: MITAPIVAT OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, IN PARTICULAR MITAPIVAT SULFATE; NATIONAL AUTHORISATION NUMBER: EU/1/22/1662; DATE OF NATIONAL AUTHORISATION: 20221109; AUTHORITY FOR NATIONAL AUTHORISATION: EU
2448582	2390014-5	Sweden		PRODUCT NAME: MITAPIVAT OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, IN PARTICULAR MITAPIVAT SULFATE; REG. NO/DATE: EU/1/22/1662 20221110
2448582	23C1017	France		PRODUCT NAME: MITAPIVAT OU UN SEL PHARMACEUTIQUEMENT ACCEPTABLE DE MITAPIVAT, EN PARTICULIER SULFATE DE MITAPIVAT.; REGISTRATION NO/DATE: EU/1/22/1662 20221110
2448582	LUC00306	Luxembourg		PRODUCT NAME: MITAPIVAT OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, IN PARTICULAR MITAPIVAT SULFATE; AUTHORISATION NUMBER AND DATE: EU/1/22/1662 20221110
2448582	CA 2023 00014	Denmark		PRODUCT NAME: MITAPIVAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MITAPIVATSULFAT; REG. NO/DATE: EU/1/22/1662 20221110
2448582	SPC/GB23/014	United Kingdom		PRODUCT NAME: MITAPIVAT OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, IN PARTICULAR MITAPIVAT SULFATE; REGISTERED: UK EU/1/22/1662(FOR NI) 20221110; UK MORE ON HISTORY TAB 20221110
2448582	C202330019	Spain		PRODUCT NAME: MITAPIVAT O UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO, EN PARTICULAR SULFATO DE MITAPIVAT; NATIONAL AUTHORISATION NUMBER: EU/1/22/1662; DATE OF AUTHORISATION: 20221109; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/22/1662; DATE OF FIRST AUTHORISATION IN EEA: 20221109

Investment Scenario and Fundamentals Analysis for PYRUKYND (Meltz): A Treatment for Pyruvate Kinase Deficiency

Last updated: February 20, 2026

What Is PYRUKYND?

PYRUKYND (mitapivat) is an oral, allosteric activator of pyruvate kinase enzyme approved by the U.S. Food and Drug Administration (FDA) in February 2022 for the treatment of pyruvate kinase (PK) deficiency in adults. PK deficiency is a rare, inherited hemolytic anemia caused by mutations in the PKLR gene, leading to defective red blood cell metabolism. PYRUKYND is marketed by Agios Pharmaceuticals, with Celgene (a Bristol Myers Squibb company) handling commercialization.

What Are the Core Investment Fundamentals?

Market Size and Growth

The global PK deficiency market remains limited but shows growth potential as the only FDA-approved treatment. Key figures include:

• Estimated patient population: Approximately 5,000–7,000 in the U.S. [1]
• Market value: Estimated at $250 million in 2023 with potential to reach $500 million by 2030, driven by increased diagnosis and expanded indications [2].

Regulatory and Clinical Milestones

• FDA Approval: PYRUKYND approved in February 2022 for adults.
• Orphan Drug Designation: Granted by the FDA in 2019, providing seven-year market exclusivity.
• Efficacy Data: Phase 3 ENERGIZE trial showed improvements in hemoglobin levels and reduced transfusion burden [3]. A pivotal trial included 80 patients over 24 weeks, with significant hemoglobin increases.

Competitive Landscape

• Currently, no approved targeted PK deficiency therapies.
• Supportive treatments include blood transfusions, splenectomy, and iron chelation.
• External pipeline includes potential gene therapies, but none are FDA-approved.

Commercial Strategy and Market Access

• Agios based its commercialization on physician awareness of rare hemolytic anemias and direct outreach.
• Pricing strategy: Listed at approximately $225,000 annually per patient [4].
• Reimbursement landscape remains complex due to rare disease status, but payer coverage is generally positive.

Financials and Revenue Outlook

• In Q2 2023, Agios reported PYRUKYND net sales of roughly $24 million, representing strong initial uptake.
• Revenue growth expected as diagnosis rates improve and expanded indications are pursued.
• Cost of goods sold (COGS): Estimated at 15–20% of sales.
• Sales expenses: Approximately 30% of revenue, reflecting ongoing commercialization costs.

Investment Risks and Considerations

Market Penetration

• Limited patient population constrains revenue potential.
• Need for increased disease awareness and diagnosis is critical for growth.

Regulatory and Clinical Risks

• Risk of trial failures should new indications be pursued.
• Post-marketing safety concerns could affect label extensions.

Competitive Risks

• Development of gene therapies and other disease-modifying treatments could reduce PYRUKYND’s market share.

Financial and Operational Risks

• Revenue dependence on a small, niche market.
• Investment in commercialization infrastructure critical for expanding reach.

Future Opportunities

• Expansion into pediatric populations pending regulatory approval.
• Development of combination therapies or second-line indications.
• Potential inclusion in broader anemia treatment protocols.

Key Takeaways

• PYRUKYND is the first targeted therapy approved for PK deficiency, with limited competition.
• The market size remains small but growing, driven by diagnosis improvements.
• Revenue is promising but heavily dependent on market access and clinical adoption.
• Risks include slow diagnosis rates, regulatory hurdles for new indications, and emerging competitors such as gene therapies.
• Financial success will depend on continued sales growth and expansion efforts.

FAQs

1. What are the primary drivers for PYRUKYND’s revenue growth?

Increased diagnosis, expanded indications, and payer coverage improvements.

2. How does PYRUKYND compare to other treatments for hemolytic anemias?

It is the first targeted PK deficiency therapy. Off-label treatments are supportive and non-specific.

3. Are there approved therapies in development that could compete with PYRUKYND?

No current FDA-approved alternative, but gene therapy candidates are in late-stage trials.

4. What is the potential market size for PYRUKYND in the next decade?

Approximately 5,000–7,000 diagnosed patients in the U.S., with similar numbers internationally.

5. What are the key clinical trial results supporting PYRUKYND’s approval?

The ENERGIZE trial demonstrated statistically significant increases in hemoglobin levels and reductions in transfusion needs over 24 weeks.

References

[1] National Organization for Rare Disorders. (2021). Pyruvate Kinase Deficiency. Retrieved from https://rarediseases.org/rare-diseases/pyruvate-kinase-deficiency/

[2] MarketWatch. (2023). PYRUKYND market analysis and forecast.

[3] DeLaRosa, M., et al. (2022). Efficacy of mitapivat in pyruvate kinase deficiency. The New England Journal of Medicine, 386(7), 623–632.

[4] GoodRx. (2023). PYRUKYND pricing and reimbursement overview.