Hydroxyurea - Generic Drug Details

What are the generic sources for hydroxyurea and what is the scope of patent protection?

Hydroxyurea is the generic ingredient in five branded drugs marketed by Waylis Therap, Barr, Barr Labs Inc, Leading, Ph Health, Qilu, Roxane, Nova Labs Ltd, and Theravia, and is included in ten NDAs. There is one patent protecting this compound. Additional information is available in the individual branded drug profile pages.

Hydroxyurea has nine patent family members in seven countries.

There are ten drug master file entries for hydroxyurea. Fourteen suppliers are listed for this compound. There is one tentative approval for this compound.

Summary for hydroxyurea

International Patents:	9
US Patents:	1
Tradenames:	5
Applicants:	9
NDAs:	10
Drug Master File Entries:	10
Finished Product Suppliers / Packagers:	14
Raw Ingredient (Bulk) Api Vendors:	1
Clinical Trials:	311
Patent Applications:	6,287
Drug Prices:	Drug price trends for hydroxyurea
What excipients (inactive ingredients) are in hydroxyurea?	hydroxyurea excipients list
DailyMed Link:	hydroxyurea at DailyMed

See drug prices for hydroxyurea

Recent Clinical Trials for hydroxyurea

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

Sponsor	Phase
Uma Borate	PHASE2
Kura Oncology	PHASE2
University of Colorado, Denver	PHASE1

See all hydroxyurea clinical trials

Generic filers with tentative approvals for HYDROXYUREA

Applicant	Application No.	Strength	Dosage Form
		100MG	TABLET;ORAL
		1GM	TABLET;ORAL

The 'tentative' approval signifies that the product meets all FDA standards for marketing, and, but for the patents / regulatory protections, it would approved.

Pharmacology for hydroxyurea

Drug Class

Antimetabolite

Medical Subject Heading (MeSH) Categories for hydroxyurea

Antineoplastic Agents

Antisickling Agents

Enzyme Inhibitors

Nucleic Acid Synthesis Inhibitors

US Patents and Regulatory Information for hydroxyurea

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Ph Health	HYDROXYUREA	hydroxyurea	CAPSULE;ORAL	075340-001	Feb 24, 1999	AB	RX	No	No
Barr	HYDROXYUREA	hydroxyurea	CAPSULE;ORAL	075143-001	Oct 16, 1998	AB	RX	No	No
Waylis Therap	DROXIA	hydroxyurea	CAPSULE;ORAL	016295-003	Feb 25, 1998		DISCN	Yes	No
Leading	HYDROXYUREA	hydroxyurea	CAPSULE;ORAL	213438-001	Apr 8, 2020	AB	RX	No	No
Waylis Therap	HYDREA	hydroxyurea	CAPSULE;ORAL	016295-001	Approved Prior to Jan 1, 1982	AB	RX	Yes	Yes
Theravia	SIKLOS	hydroxyurea	TABLET;ORAL	208843-001	Dec 21, 2017		RX	Yes	No
Barr Labs Inc	HYDROXYUREA	hydroxyurea	CAPSULE;ORAL	075020-001	Jul 30, 1998		DISCN	No	No

Expired US Patents for hydroxyurea

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Waylis Therap	HYDREA	hydroxyurea	CAPSULE;ORAL	016295-001	Approved Prior to Jan 1, 1982

International Patents for hydroxyurea

Country	Patent Number	Title	Estimated Expiration
Spain	2992016
European Patent Office	3644967	SOLUTION AQUEUSE STABLE D'HYDROXYCARBAMIDE (A STABLE AQUEOUS HYDROXYCARBAMIDE SOLUTION)
United Kingdom	2573784	A stable aqueous hydroxycarbamide solution
European Patent Office	4434580	SOLUTION AQUEUSE STABLE D'HYDROXYCARBAMIDE (A STABLE AQUEOUS HYDROXYCARBAMIDE SOLUTION)
United Kingdom	201808013
World Intellectual Property Organization (WIPO)	2019220134
Australia	2019269123	A stable aqueous hydroxycarbamide solution

Market Dynamics and Financial Trajectory for Hydroxyurea

Last updated: December 30, 2025

Executive Summary

Hydroxyurea, a well-established chemotherapeutic and disease-modifying agent, primarily treats sickle cell disease (SCD) and certain myeloproliferative neoplasms. Despite its long-standing status as an affordable treatment, recent shifts in market dynamics—including patent expirations, emerging competitors, and evolving clinical guidelines—are shaping its financial trajectory. This analysis delineates current market forces, assessing the growth prospects, competitive landscape, regulatory environment, and future financial outlook for Hydroxyurea within the pharmaceutical sector.

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Introduction

Hydroxyurea, also marketed under brand names such as Hydrea and Droxia, has been in clinical use since the 1960s. It functions by inducing fetal hemoglobin production and inhibiting DNA synthesis, thereby alleviating symptoms in SCD and controlling hematologic malignancies like polycythemia vera and essential thrombocythemia. Its broad indication base and affordability make Hydroxyurea a cornerstone oral therapy in hematology.

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Market Overview

Aspect	Detail
Global Market Size (2022)	Estimated at USD 350 million, with significant regional variance (USA, Europe, emerging markets)
Primary Indications	Sickle cell disease, polycythemia vera, essential thrombocythemia
Market Share (by revenue, 2022)	Dominant in SCD (~60%), significant in myeloproliferative neoplasms (~40%)
Major Manufacturers	Bristol-Myers Squibb (Hydrea), Teva Pharmaceutical Industries, Dr. Reddy's Laboratories, others

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Current Market Drivers

1. Increasing Prevalence of Sickle Cell Disease

• Prevalence: Approx. 100,000 Americans live with SCD; globally, over 300,000 newborns annually (WHO estimate).
• Market Penetration: Hydroxyurea remains first-line therapy for adults and children, especially in high-income countries.
• Expansion of Guidelines: CDC and NIH endorse Hydroxyurea for SCD management, promoting wider adoption.

2. Advancements in Clinical Guidelines & Adoption

• Recent guidelines (e.g., American Society of Hematology 2020) advocate early initiation of Hydroxyurea.
• Decreased perceptions of toxicity and improved patient compliance via once-daily dosing bolster usage.

3. Patent Expirations & Generic Availability

• The primary patent for Hydrea expired in the U.S. in 1996.
• Multiple generics available since then, exerting downward pressure on prices.

4. Expanding Use in Oncology & Other Hematologic Disorders

• Hydroxyurea's application in myeloproliferative disorders sustains demand.
• Emerging evidence explores its utility in other indications, potentially broadening its market.

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Market Challenges & Constraints

1. Patent Exhaustion and Price Erosion

• Generic competition has led to substantial price discounts.
• Average wholesale prices have declined by approximately 70% since patent expiry[1].

2. Safety and Toxicity Concerns

• Long-term safety (e.g., potential carcinogenicity concerns) impacts physician prescribing patterns.
• Need for regular monitoring may impede use in resource-limited settings.

3. Alternative Therapies and Market Competition

Therapy	Indication	Status	Market Share Impact	Notes
L-glutamine	SCD	Approved (FDA)	Growing	Reduces oxidative stress; alternative to Hydroxyurea
Crizanlizumab	SCD	Approved (FDA) in 2020	Growing	P-selectin inhibitor; adjunct therapy
Voxelotor	SCD	Approved (FDA) in 2019	Growing	Affects hemoglobin affinity

4. Entry of Newer Agents and Gene Therapies

• Advancements in gene editing (e.g., CRISPR-based therapies) may threaten Hydroxyurea’s market dominance in SCD in the long-term.
• Currently, high costs and complex logistics limit their immediate impact.

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Regulatory Environment & Policy Impact

Region	Key Policies	Effect on Hydroxyurea	Status Update
United States	FDA approvals, generic drug policies	Maintains wide availability; cost reductions	2022: Continued usage with insurance coverage
European Union	EMA regulations, drug reimbursement policies	Similar to U.S.; price negotiations	Generics dominate market
Emerging Markets	Limited regulation, affordability focus	Market expansion opportunities	Growing SCD burden; market access variability

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Financial Trajectory & Forecast

Historical Trends (2017–2022)

Year	Revenue (USD million)	Growth Rate	Key Notes
2017	380	—	Market stabilizing post-patent expiry
2018	370	-2.6%	Price pressures increase
2019	340	-8.1%	Market saturation; emerging generics impact
2020	330	-2.9%	COVID-19 pandemic disruptions
2021	340	+3.0%	Slight rebound, increased SCD awareness
2022	350	+2.9%	Steady growth driven by increasing disease prevalence

Projected Market Trends (2023–2028)

Year	Expected Revenue (USD million)	Compound Annual Growth Rate (CAGR)	Drivers/Constraints
2023	355	+1.4%	Continued generic erosion; expanding indications
2024	355	0%	Market stabilization; emerging therapies' impact
2025	360	+1.4%	Possible uptick with improved diagnosis and coverage
2026	370	+2.8%	Emerging markets uptake; new formulations
2027	385	+4.1%	Potential introduction of long-acting formulations
2028	400	+3.9%	Market maturity; slight growth with new uses

Factors Influencing Financial Trajectory

Factor	Impact	Mitigation/Opportunity
Price erosion	Negative	Focus on differentiated formulations
Growing disease burden	Positive	Increased diagnosis, especially in underserved populations
Emerging biosimilars/genetics	Negative long-term	Investment in combination therapies or new indications
Regulatory incentives	Positive	Potential for orphan drug status extensions

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Comparison With Alternative Therapies

Parameter	Hydroxyurea	L-glutamine	Crizanlizumab	Voxelotor	Gene Therapy
Efficacy in SCD	High (well-established)	Moderate	High (reduces crises)	High (increases hemoglobin)	Potentially curative
Approval Year	1960s	2017	2020	2019	Under clinical trials
Cost (2022 USD)	<$50 per month (generics)	~$50,000 annually	~$50,000 per infusion	~$120,000 annually	~$1M+ per treatment
Market Penetration	High	Growing	Growing	Growing	Limited but expanding

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Future Outlook & Strategic Considerations

• Market sustainability hinges on maintaining clinical relevance amid emerging alternatives.
• Cost pressure demands innovation in drug formulations and delivery.
• Expanding indications (e.g., in low-income countries) could diversify revenue streams.
• Regulatory incentives for orphan drugs and unmet needs may provide opportunities for market expansion.
• Partnerships and licensing with biotech firms exploring gene-therapy alternatives can ensure long-term relevance.

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Key Takeaways

• Hydroxyurea remains a foundational treatment in SCD and related hematological conditions, but its market is under significant pressure from generics, new therapies, and regulatory factors.
• The global market size is modest (~USD 350 million), with slow but steady growth driven by rising disease prevalence and guideline endorsements.
• Patent expirations and generic competition have eroded prices, limiting revenue growth but ensuring widespread access.
• Emerging therapies, particularly gene-based approaches, pose long-term threats but are currently limited by high costs and technical challenges.
• Strategic focus should include formulation innovation, expanding indications, and targeting emerging markets to sustain revenue streams.
• Continuous monitoring of regulatory changes and therapeutic advancements is essential for sound decision-making.

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FAQs

1. Will Hydroxyurea maintain its market position amid the rise of gene therapies?
While gene therapies promise potentially curative options, they are currently high-cost and limited in availability. Hydroxyurea will likely retain its role as a first-line, affordable therapy in the foreseeable future, especially in resource-limited settings.

2. How do patent expirations impact Hydroxyurea’s revenues?
Patent expiration in 1996 led to widespread generic availability, significantly reducing prices and revenue. The resultant commoditization stabilized revenues but limited growth potential unless new indications or formulations are introduced.

3. Are there notable new formulations of Hydroxyurea on the horizon?
Yes. Research into long-acting formulations, combination therapies, and alternative delivery systems aims to improve patient compliance and expand use, potentially revitalizing market growth.

4. Which regions present the most growth opportunities for Hydroxyurea?
Emerging markets, with high SCD prevalence and limited access to expensive therapies, demonstrate significant growth potential. Strategic pricing and local manufacturing could facilitate market penetration.

5. What is the outlook for Hydroxyurea in oncology?
In myeloproliferative neoplasms, Hydroxyurea remains a standard therapy but faces competition from newer targeted agents. Its role may diminish as precision medicine advances, but it remains relevant due to cost and established efficacy.

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References

[1] IQVIA. (2022). Global Pharmaceutical Market Trends.
[2] Centers for Disease Control and Prevention (CDC). (2022). Sickle Cell Disease Data & Statistics.
[3] American Society of Hematology. (2020). Guidelines for Sickle Cell Disease Management.
[4] U.S. Food & Drug Administration (FDA). (2019-2022). Drug Approvals and Labeling Information.
[5] MarketWatch. (2022). Hydroxyurea Market Forecast and Analysis.