PYRUKYND Drug Patent Profile

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

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

This drug has one hundred and ninety-one 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 will be eligible for patent challenges on February 17, 2026. This date may extended up to six months if a pediatric exclusivity extension is applied to the drug's patents.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be November 21, 2038. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for PYRUKYND

International Patents:	191
US Patents:	9
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	33
Drug Prices:	Drug price information for PYRUKYND
What excipients (inactive ingredients) are in PYRUKYND?	PYRUKYND excipients list
DailyMed Link:	PYRUKYND at DailyMed

See drug prices for PYRUKYND

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

NDA:

216196

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.

Pharmacology for PYRUKYND

Drug Class	Pyruvate Kinase Activator
Mechanism of Action	Cytochrome P450 2B6 Inducers Cytochrome P450 2C19 Inducers Cytochrome P450 2C8 Inducers Cytochrome P450 2C9 Inducers Cytochrome P450 3A Inducers P-Glycoprotein Inhibitors Pyruvate Kinase Activators UGT1A1 Inducers

US Patents and Regulatory Information for PYRUKYND

PYRUKYND is protected by nine US patents and two 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
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
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
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-001	Feb 17, 2022		RX	Yes	No
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-002	Feb 17, 2022		RX	Yes	No

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:

Australia

Brazil

Canada

China

Croatia

Denmark

Eurasian Patent Organization

European Patent Office

Finland

Hungary

Israel

Japan

Lithuania

Mexico

Moldova, Republic of

Morocco

Philippines

Poland

Portugal

San Marino

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 PYRUKYND around the world.

Country	Patent Number	Title	Estimated Expiration
Japan	7320339
Costa Rica	20170071
South Korea	101850813
Israel	276414	אקטיבטור לקינאז של פירובאט לשימוש בטיפול (Pyruvate kinase activators for use in therapy)
South Korea	20140028008	PYRUVATE KINASE ACTIVATORS FOR USE IN THERAPY
Israel	276414

Supplementary Protection Certificates for PYRUKYND

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2448582	CR 2023 00014	Denmark		PRODUCT NAME: MITAPIVAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MITAPIVATSULFAT; REG. NO/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	2023C/517	Belgium		PRODUCT NAME: MITAPIVAT OU UN SEL PHARMACEUTIQUEMENT ACCEPTABLE DE CELUI-CI, EN PARTICULIER LE SULFATE DE MITAPIVAT; AUTHORISATION NUMBER AND DATE: EU/1/22/1662 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
2448582	301230	Netherlands		PRODUCT NAME: MITAPIVAT, DAN WEL EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN, IN HET BIJZONDER MITAPIVATSULFAAT; REGISTRATION NO/DATE: EU/1/22/1662 20221110
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

Market Dynamics and Financial Trajectory for Pyrukynd

Last updated: July 27, 2025

Introduction

Pyrukynd (mitapivat) has emerged as a notable development within the pharmaceutical landscape, primarily targeting rare hematologic conditions such as pyruvate kinase (PK) deficiency. As a first-in-class transformative therapy, Pyrukynd’s commercial journey is shaped by evolving clinical data, regulatory milestones, market demand, and competitive dynamics. This analysis offers a comprehensive assessment of Pyrukynd’s current market position, growth prospects, and the underpinnings influencing its financial trajectory.

Product Overview and Indication Landscape

Pyrukynd, developed by Agios Pharmaceuticals, is an oral, allosteric activator of red blood cell pyruvate kinase aimed at patients with PK deficiency — a rare, inherited hemolytic anemia caused by mutations in the PKLR gene. The unmet medical need in this niche, compounded by limited alternative therapies, positions Pyrukynd as a potentially pivotal therapeutic agent.

The FDA approved Pyrukynd in February 2022 for the treatment of hemolytic anemia in adult patients with PK deficiency, marking a significant milestone. The drug’s approval expanded its commercial scope domestically, with subsequent regulatory reviews anticipated globally, particularly in European markets.

Market Dynamics

1. Unmet Medical Need and Market Size

PK deficiency affects approximately 5,000-10,000 individuals in the United States and Europe, with many patients experiencing chronic anemia, jaundice, and potential complications such as iron overload. The rarity classifies it as an ultra-rare disease, resulting in high-value, niche market dynamics. The limited availability of targeted therapies amplifies the clinical importance of Pyrukynd, accelerating demand among specialist physicians and patient advocacy groups.

2. Competitive Landscape

Currently, management primarily involves supportive care, including blood transfusions and splenectomy. No approved disease-modifying therapies exist outside Pyrukynd, granting exclusivity that enhances market capture prospects. However, other pharmaceutical entities are exploring gene therapy and alternative small molecules, which could influence the competitive environment over the next decade.

3. Regulatory and Reimbursement Environment

Regulatory agencies globally are demonstrating increasing openness to orphan drugs, often offering accelerated approval pathways, as evidenced by Pyrukynd’s FDA approval under priority review. Reimbursement negotiations hinge on demonstrating clinical benefits, cost-effectiveness, and real-world efficacy, factors that will impact adoption and market penetration rates.

4. Clinical Data and Label Expansion

The ongoing phase 3 PYRUKYND-301 trial aims to expand Pyrukynd’s label beyond initial indications, possibly into pediatric populations or broader anemia-related conditions. Positive trial outcomes could substantially enlarge the target market and improve health economic profiles, thereby bolstering the financial trajectory.

5. Market Penetration and Access Strategies

Pursuing direct sales efforts, partnering with specialty pharmacies, and engaging patient advocacy groups are crucial for market expansion. Awareness campaigns, clinician education, and demonstrating long-term value through real-world evidence will influence uptake rates.

Financial Trajectory Analysis

1. Revenue Generation and Sales Outlook

Since its 2022 approval, Pyrukynd’s revenue projections are driven by initial uptake in the US, where specialty pharmacies and hematology clinics serve as primary sales channels. Industry analyst estimates suggest revenues reaching approximately $100-$150 million in the first 3 years, contingent on market penetration and approval in European territories.

2. Pricing Strategies

Orphan drugs often command high prices due to limited patient populations and development costs. Pyrukynd’s pricing is expected to be in the range of $100,000 to $250,000 annually per patient, with discounts and value-based agreements influencing net revenue. Pricing negotiations will be influenced by comparative efficacy data and payer dynamics.

3. Cost Structure and Investment

Manufacturing costs are relatively low for small-molecule drugs; however, substantial investments are necessary for clinical trials, regulatory filings, and commercialization activities. Marketing expenditures targeting specialist physicians and patient advocacy are integral to market success.

4. Market Share Evolution

Initial market share is anticipated to be modest but growing rapidly as prescription volumes increase. The potential for phase 3 trial success and subsequent label expansions could accelerate market share capture and boost long-term revenues.

5. Long-Term Revenue Potential

With successful phase 3 trials, label broadening, and possible inclusion of pediatric patients, Pyrukynd could see revenues reaching hundreds of millions annually within a decade. Additional indications in related hemolytic anemias or metabolic disorders might further diversify revenue streams.

Key Factors Affecting Financial Performance

• Regulatory approvals in international markets: Facilitating broader access and higher revenues.
• Clinical trial outcomes: Influencing label expansion and payer acceptance.
• Market penetration strategies: Impacting speed and extent of adoption among hematologists.
• Pricing and reimbursement negotiations: Ultimately determining net revenue.
• Competitive innovations: Potential advancements in gene therapy could threaten market share.

Strategic Considerations for Stakeholders

To maximize financial outcomes, stakeholders should focus on accelerating clinical development, engaging in early payer discussions, and investing in educational initiatives aimed at clinicians and patients. Collaboration with health authorities for managed entry agreements and outcome-based pricing will enhance access and revenue growth.

Conclusion

Pyrukynd’s introduction signifies a paradigm shift in managing PK deficiency, offering a disease-modifying therapy that addresses a critical unmet need. The market dynamics are favorable, bolstered by regulatory support and limited competition, with significant upside potential—contingent on clinical success and strategic market deployment. Financially, Pyrukynd is positioned for substantial growth, provided that commercialization strategies align with clinical progress and health economic validation.

Key Takeaways

• Pyrukynd benefits from a high unmet need in an ultra-rare disease, supporting rapid initial adoption.
• Regulatory momentum and label expansion prospects will be pivotal to scaling revenues.
• Market penetration depends on effective clinician engagement, payer negotiations, and patient awareness.
• Pricing strategies reflect the orphan drug premium, influencing net revenues.
• Long-term success hinges on clinical data, competitive landscape evolution, and global regulatory approvals.

FAQs

1. What distinguishes Pyrukynd from existing treatments for PK deficiency?
Pyrukynd is the first approved disease-modifying, oral therapy targeting the underlying enzyme deficiency in PK deficiency, unlike supportive measures such as transfusions and splenectomy.

2. How does Pyrukynd’s recent approval impact its market outlook?
Approval provides a clear regulatory pathway and initial revenue stream. It enhances credibility among clinicians and payers, promising robust market penetration especially if subsequent approvals and label expansions materialize.

3. What are the main risks to Pyrukynd’s financial success?
Risks include failure in ongoing or future clinical trials, unfavorable reimbursement negotiations, emergence of competitors or gene therapies, and limited awareness in broader healthcare settings.

4. How might global regulatory trends influence Pyrukynd’s market expansion?
Increasing orphan drug incentives and expedited review pathways in Europe and Asia could accelerate international commercialization, expanding revenue opportunities.

5. What future therapeutic developments could challenge Pyrukynd’s market dominance?
Gene therapy approaches targeting PK deficiency and other disease modifying strategies in development could threaten long-term market share if proven safe and effective.

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Sources:
[1] Agios Pharmaceuticals Press Release, February 2022.
[2] FDA Approval Document for Pyrukynd.
[3] Market Research Reports on Rare Hematologic Disorders.
[4] Industry Analyst Estimates (2023).