Mitapivat sulfate - Generic Drug Details

What are the generic sources for mitapivat sulfate and what is the scope of freedom to operate?

Mitapivat sulfate is the generic ingredient in one branded drug marketed by Agios Pharms Inc and is included in one NDA. There are nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Mitapivat sulfate has one hundred and ninety patent family members in forty-six countries.

One supplier is listed for this compound.

Summary for mitapivat sulfate

International Patents:	190
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	14
DailyMed Link:	mitapivat sulfate at DailyMed

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

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 mitapivat sulfate

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

Sponsor	Phase
University Health Network, Toronto	PHASE2
Agios Pharmaceuticals, Inc.	PHASE2
EuroBloodNet Association	Phase 2

See all mitapivat sulfate clinical trials

Pharmacology for mitapivat sulfate

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

Anatomical Therapeutic Chemical (ATC) Classes for mitapivat sulfate

B06AX	Other hematological agents
B06A	OTHER HEMATOLOGICAL AGENTS
B06	OTHER HEMATOLOGICAL AGENTS
B	Blood and blood forming organs

US Patents and Regulatory Information for mitapivat sulfate

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	216196-002	Feb 17, 2022		RX	Yes	No
Agios Pharms Inc	PYRUKYND	mitapivat sulfate	TABLET;ORAL	216196-002	Feb 17, 2022		RX	Yes	No
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	216196-003	Feb 17, 2022		RX	Yes	Yes

International Patents for mitapivat sulfate

Country	Patent Number	Title	Estimated Expiration
Peru	20120693	COMPUESTOS HETEROCICLOS COMO MODULADORES DE LA PIRUVATO CINASA M2 (PKM2)
Serbia	65215	AKTIVATORI PIRUVAT KINAZE ZA UPOTREBU U TERAPIJI (PYRUVATE KINASE ACTIVATORS FOR USE IN THERAPY)
Portugal	2704721
World Intellectual Property Organization (WIPO)	2019104134
European Patent Office	4285904

Supplementary Protection Certificates for mitapivat sulfate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
2448582	122023000025	Germany		PRODUCT NAME: MITAPIVAT ODER EIN PHARMAZEUTISCH AKZEPTABLES SALZ DAVON, INSBESONDERE MITAPIVATSULFAT; REGISTRATION NO/DATE: EU/1/22/1662 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	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 MITAPIVAT SULFATE

Last updated: July 29, 2025

Introduction

Mitapivat sulfate, a novel oral small-molecule activator of pyruvate kinase (PKR), has garnered significant attention within the pharmaceutical landscape due to its potential to transform treatment paradigms for rare hematological disorders, notably pyruvate kinase deficiency (PKD). As the drug advances through clinical trials and regulatory pathways, understanding the market dynamics and financial trajectory is essential for stakeholders poised to capitalize on its commercialization potential.

Market Overview

Mitapivat sulfate addresses a high unmet medical need in PKD, a rare inherited disorder characterized by chronic hemolytic anemia stemming from defective erythrocyte energy metabolism. The global orphan drug market, valued at approximately USD 140 billion in 2022, figures prominently in the prospective commercial success of mitapivat*, owing to regulatory incentives and a focused patient population. The increasing recognition of PKD's burden and pending approvals offer a promising landscape for the drug’s market entry.

Regulatory Landscape and Development Status

Eli Lilly and AbbVie have pioneered in advancing mitapivat’s clinical development, with pivotal Phase III trials exhibiting promising efficacy data. The FDA granted Breakthrough Therapy designation for mitapivat in PKD (as of 2021), accelerating its review process. As FDA review progresses, potential approval timelines forecast availability within two years, contingent upon successful trial outcomes. This regulatory momentum accelerates market entry and influences the competitive landscape.

Market Drivers

1. Unmet Clinical Need: PKD's lack of approved targeted therapies drives demand for mitapivat. Current management relies mainly on supportive care, such as transfusions and splenectomy, with no disease-modifying options.
2. Efficacy and Safety Data: Early-phase studies have demonstrated significant increases in hemoglobin levels and reductions in hemolysis metrics, corroborating clinical benefits. The favorable safety profile enhances market acceptance potential.
3. Orphan Drug Incentives: Regulatory exclusivities, reduced development costs, and sponsorship opportunities lower market entry barriers.
4. Growing Awareness and Diagnosis: Advances in genetic testing and increased disease prevalence awareness expand the diagnosed patient pool.

Market Challenges

• Pricing and Reimbursement: High treatment costs typical of orphan drugs may challenge payer acceptance. Demonstrating cost-effectiveness remains critical.
• Competition: While currently limited, potential entrants developing alternative therapies or gene editing approaches could impact mitapivat’s market share.
• Manufacturing and Supply Chain: Ensuring consistent clinical-grade manufacturing will be pivotal as demand materializes.

Financial Trajectory

1. Revenue Projections

Early estimations, based on comparable orphan drugs and global PKD prevalence estimates (approximately 3000–10,000 patients in the U.S. alone), suggest robust initial uptake. A conservative projection anticipates:

• Year 1: Launch phase, capturing 10–15% of diagnosed patients, generating USD 300–500 million globally, considering premium pricing of USD 100,000–150,000 annually per patient.
• Year 3–5: Expanding orphan drug coverage, with market penetration rising to 40–60%, resulting in USD 1–1.5 billion in annual revenues globally.

2. Cost Structure

• Research and Development: Continued investment into post-trial studies and real-world evidence collection.
• Manufacturing: Scaling production capabilities aligned with demand forecasts.
• Commercialization: Marketing, regulatory compliance, and distribution costs will influence overall profitability.

3. Profitability Outlook

Given the typical high-margin profiles of orphan drugs, gross margins of 70–80% are plausible. Early revenue streams, coupled with limited competition, reinforce a strong financial outlook contingent upon successful regulatory approval and payer acceptance.

Market Penetration and Growth Strategies

To optimize financial outcomes, companies may adopt strategies such as:

• Early engagement with payers to secure favorable reimbursement agreements.
• Geographic expansion into European, Asian, and emerging markets where PKD diagnosis is underrecognized.
• Post-approval label expansions, including combination therapies or broader indications.
• Investment in patient advocacy networks to foster early diagnosis and adherence.

Competitive Landscape

Currently, mitapivat’s primary competitor remains supportive care, with emerging biological agents, gene therapies, and other small molecules under investigation that could threaten market share. Early market dominance hinges upon swift regulatory approval, strong clinical data, and effective commercialization.

Regulatory and Market Risks

Uncertainties include FDA and EMA approval timelines, potential adverse event disclosures, and reimbursement hurdles. Market prices set above value thresholds risk reimbursement rejection, emphasizing the importance of health-economic evidence development.

Conclusion

Mitapivat sulfate embodies a transformative opportunity within the orphan drug and hematology segments. The market dynamics driven by unmet medical need, favorable regulatory pathways, and institutional investment underpin a burgeoning financial trajectory. Strategic positioning, early access negotiations, and post-market evidence generation will determine long-term revenue potential.

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Key Takeaways

• Market Potential: Mitapivat's innovative mechanism promises significant adoption within the rare PKD market, with estimated revenues reaching USD 1–1.5 billion globally within a few years post-launch.
• Regulatory Progress: Pending FDA and EMA approvals are critical inflection points; Breakthrough Therapy designation expedites pathway prospects.
• Commercial Strategy: Early payer engagement, geographic expansion, and label broadening are vital to maximizing financial gains.
• Challenges: Price setting, reimbursement hurdles, and emerging competition require proactive mitigation strategies.
• Investment Outlook: With high margins typical of orphan drugs and a focused patient population, mitapivat’s financial trajectory is favorable—assuming successful commercialization and market acceptance.

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FAQs

Q1: What is the expected timeline for FDA approval of mitapivat sulfate?
A: Based on current clinical data and regulatory review cycles, FDA approval is anticipated within two years, provided ongoing trials confirm efficacy and safety.

Q2: How large is the potential patient population for mitapivat in the U.S.?
A: Approximately 3,000 to 10,000 diagnosed PKD patients are estimated in the U.S., representing the initial core market for mitapivat.

Q3: What are the primary financial challenges associated with launching mitapivat?
A: Key challenges include setting sustainable pricing that balances reimbursement with profitability, overcoming payer reluctance, and ensuring manufacturing scalability.

Q4: How might competition impact mitapivat’s market share?
A: While current competition is limited, future entrants developing gene therapies or alternative small molecules could threaten market dominance, necessitating continuous innovation and differentiation.

Q5: What additional indications could enhance mitapivat's market viability?
A: Beyond PKD, investigational evidence suggests potential utility in other hemolytic anemias, which could broaden the drug’s application and revenue streams.

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References

1. [1] Global Orphan Drug Market Analysis, 2022.
2. [2] ClinicalTrials.gov. Mitapivat Trials for PKD.
3. [3] FDA. Breakthrough Therapy Designation for Mitapivat.
4. [4] Market projections for rare hematologic disorders, 2023.
5. [5] Pricing and reimbursement insights for orphan drugs, 2022.