PYRUKYND Drug Patent Profile

Which patents cover Pyrukynd, 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.

This drug has one hundred and eighty-six 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:	186
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

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
Hungary	E063541
Australia	2024200724	Crystalline forms of N-(4-(4-(cyclopropylmethyl) piperazine-1-carbonyl)phenyl)quinoline-8-sulfonamide
Serbia	64592	KRISTALNE FORME N-(4-(4-(CIKLOPROPILMETIL)PIPERAZIN-1-KARBONIL)FENIL) HINOLIN-8-SULFONAMIDA (CRYSTALLINE FORMS OF N-(4-(4-(CYCLOPROPYLMETHYL) PIPERAZINE-1-CARBONYL)PHENYL)QUINOLINE-8-SULFONAMIDE)

Supplementary Protection Certificates for PYRUKYND

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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	122023000025	Germany		PRODUCT NAME: MITAPIVAT ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON, INSBESONDERE MITAPIVATSULFAT; REGISTRATION NO/DATE: EU/1/22/1662 20221109
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

Market Dynamics and Financial Trajectory for PYRUKYND

Last updated: July 4, 2025

Introduction

PYRUKYND, the brand name for mitapivat, has emerged as a pivotal innovation in treating rare hemolytic anemias, particularly pyruvate kinase (PK) deficiency. Developed by Agios Pharmaceuticals, this oral activator of pyruvate kinase gained FDA approval in February 2022, marking a significant milestone for patients with limited treatment options. As the pharmaceutical landscape evolves, understanding PYRUKYND's market dynamics and financial path offers critical insights for investors, executives, and stakeholders navigating the high-stakes world of rare disease therapies. This analysis delves into current market forces, competitive pressures, and revenue projections, equipping business professionals with actionable intelligence.

Overview of PYRUKYND

PYRUKYND stands out as a first-in-class drug that addresses the root cause of PK deficiency by boosting red blood cell energy metabolism. This condition, affecting roughly 1 in 20,000 individuals globally, leads to chronic anemia and related complications. Agios Pharmaceuticals positioned PYRUKYND as an orphan drug, securing expedited regulatory pathways and market exclusivity that enhance its commercial appeal.

The drug's mechanism involves activating the pyruvate kinase enzyme, which improves hemoglobin levels and reduces transfusion dependence. Clinical trials, including the ACTIVATE study, demonstrated sustained efficacy, with patients experiencing significant hemolysis reduction. Priced at approximately $300,000 annually per patient in the U.S., PYRUKYND reflects the premium valuation typical of rare disease treatments, driven by its potential to transform patient outcomes and reduce healthcare burdens.

Market Dynamics

Current Market Landscape

The rare disease market, valued at over $150 billion in 2023, continues to expand at a compound annual growth rate (CAGR) of 11% through 2030, propelled by advancements in precision medicine. PYRUKYND occupies a niche within the hemolytic anemia segment, which includes conditions like sickle cell disease and thalassemia. In the U.S., it captured early market share among the estimated 3,000 to 8,000 PK deficiency patients, with initial sales reaching $50 million in its first full year post-approval.

Geographic expansion plays a key role, as Agios pursues approvals in Europe and Asia. The European Medicines Agency (EMA) granted marketing authorization in 2023, opening doors to markets where reimbursement policies favor high-value orphan drugs. However, penetration remains challenged by diagnostic limitations; only a fraction of potential patients are identified, constraining immediate demand.

Competitive Environment

Competition in the PK deficiency space is limited but intensifying. PYRUKYND currently holds a monopoly, but emerging therapies from players like Forma Therapeutics and Novartis threaten long-term dominance. For instance, Forma's FT-4202, another pyruvate kinase activator, entered Phase 3 trials in 2023, potentially eroding PYRUKYND's market share by 2026.

Broader hemolytic anemia treatments, such as Novartis' crizanlizumab for sickle cell disease, indirectly compete by diverting resources and patient focus. Pricing pressures from payers, including U.S. insurers and international health authorities, further complicate dynamics. Agios counters this through value-based pricing agreements, tying rebates to real-world outcomes, which has helped maintain PYRUKYND's accessibility in key markets.

Regulatory and External Factors

Regulatory milestones significantly influence PYRUKYND's trajectory. The FDA's orphan drug designation provides seven years of market exclusivity in the U.S., shielding it from generics until 2029. Similarly, EMA protections extend to 2030 in Europe. These factors bolster Agios' bargaining power, but evolving regulations, such as the U.S. Inflation Reduction Act's drug price negotiations, could cap revenues by introducing price controls for Medicare patients starting in 2026.

External forces, including global supply chain disruptions and economic inflation, add volatility. The COVID-19 pandemic delayed clinical trials and patient access, yet it also accelerated telehealth adoption, potentially aiding PYRUKYND's distribution. Market analysts from Evaluate Pharma project that rare disease drugs like PYRUKYND will benefit from increased R&D investments, with global spending expected to hit $200 billion by 2025.

Financial Trajectory

Revenue Projections and Performance

Agios Pharmaceuticals reported PYRUKYND net sales of $78 million in 2023, a 56% increase from the previous year, driven by expanded prescribing and international launches. Wall Street forecasts suggest revenues could climb to $300 million by 2026, assuming broader adoption and pipeline synergies. This growth hinges on Agios' $1.2 billion cash reserve, which supports ongoing marketing and R&D efforts.

The drug's financial profile benefits from high margins, with gross margins exceeding 80%, attributed to efficient manufacturing and minimal competition. However, quarterly fluctuations, such as a 15% sales dip in Q2 2023 due to inventory adjustments, highlight risks. Agios' stock, trading at a price-to-earnings ratio of 25, reflects investor optimism but remains sensitive to clinical updates and reimbursement decisions.

Investment Landscape and Challenges

Investment in PYRUKYND aligns with the biotech boom, where venture capital inflows to rare disease therapies reached $10 billion in 2023. Agios secured a $1 billion partnership with Servier in 2021 for ex-U.S. rights, providing upfront payments and milestones that fund expansion. This deal exemplifies how strategic alliances mitigate financial risks, enabling Agios to invest in adjuvant therapies.

Challenges include patent cliffs and biosimilar threats. PYRUKYND's core patents extend to 2037, offering stability, but generic entrants could emerge post-exclusivity. Financial analysts at Goldman Sachs predict a potential 20% revenue drop if competitors advance, underscoring the need for diversification. Agios is addressing this by advancing its oncology pipeline, which could generate $500 million in additional revenues by 2030.

Inflation and currency fluctuations pose further hurdles; for example, a stronger U.S. dollar reduced Agios' international earnings by 10% in 2023. Despite these, the company's debt-free balance sheet and $400 million in projected free cash flow position it for sustained growth.

Conclusion

PYRUKYND's market dynamics and financial trajectory underscore the opportunities and obstacles in the rare disease sector. As Agios navigates regulatory landscapes and competitive pressures, the drug's role in improving patient lives continues to drive its value. Business professionals must weigh these factors to inform investment and strategic decisions in an increasingly dynamic pharmaceutical market.

Key Takeaways

• PYRUKYND's sales grew 56% to $78 million in 2023, with projections reaching $300 million by 2026 amid expanding approvals.
• Orphan drug status provides market exclusivity until 2029 in the U.S., shielding revenues from immediate competition.
• High pricing and value-based agreements sustain profitability, but regulatory price caps could impact long-term growth.
• Strategic partnerships, like the Servier deal, bolster Agios' financial stability and global reach.
• Emerging competitors and external economic factors introduce volatility, necessitating diversified pipelines for risk mitigation.

FAQs

1. What is PYRUKYND and how does it work?
   PYRUKYND is an oral medication that activates the pyruvate kinase enzyme to treat PK deficiency, improving red blood cell function and reducing anemia symptoms in adults.

2. How has PYRUKYND performed financially since its launch?
   Since FDA approval in 2022, PYRUKYND generated $78 million in net sales by 2023, with analysts forecasting triple-digit growth as it gains traction in new markets.

3. What factors could influence PYRUKYND's market share?
   Regulatory approvals, competitor entries like FT-4202, and reimbursement policies will shape PYRUKYND's market position, potentially affecting its dominance in hemolytic anemia treatments.

4. What investment opportunities does PYRUKYND offer?
   Investors can capitalize on Agios' stock through its high-margin revenues and partnerships, though risks from patent expirations and economic shifts require careful monitoring.

5. How does PYRUKYND fit into the broader rare disease market?
   As a niche therapy in a $150 billion market, PYRUKYND exemplifies how targeted drugs can achieve premium pricing and growth, despite challenges in patient identification and competition.

Sources Cited

1. Agios Pharmaceuticals. (2023). Annual Report and Financial Statements. Retrieved from Agios investor relations page.
2. U.S. Food and Drug Administration. (2022). FDA Approval Letter for Mitapivat. Available at FDA.gov.
3. Evaluate Pharma. (2023). World Preview 2023, Outlook to 2030. Data on rare disease market projections.