Hydroxyurea - Generic Drug Details

What are the generic drug 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:	308
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
H. Lee Moffitt Cancer Center and Research Institute	PHASE2

See all hydroxyurea clinical trials

Generic filers with tentative approvals for HYDROXYUREA

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

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	3,968,249

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

Market Dynamics and Financial Trajectory for Hydroxyurea

Last updated: July 27, 2025

Introduction

Hydroxyurea, a well-established chemotherapeutic and disease-modifying agent, has carved a pivotal niche within the pharmaceutical landscape. Primarily used in the management of sickle cell disease (SCD) and certain malignancies, its market evolution aligns closely with therapeutic breakthroughs, regulatory policies, and rising global demand. This analysis explores the current market environment, key drivers and barriers, financial trends, and future trajectories that influence hydroxyurea’s commercial landscape.

Therapeutic Indications and Market Size

Hydroxyurea’s orphan designation and FDA approval in 1998 for sickle cell disease have sustained its position as a frontline treatment in SCD management. Its unique disease-modifying properties—including reduced frequency of vaso-occlusive crises and improved hemoglobin levels—have led to widespread adoption, particularly in regions with high disease prevalence such as sub-Saharan Africa, the Middle East, and the United States [1].

In oncology, hydroxyurea’s role extends to treating certain leukemias (chronic myeloid leukemia, melanoma) and as a chemotherapeutic adjunct. Although its position has been supplemented by targeted therapies, hydroxyurea remains economical and accessible, especially for resource-limited markets.

The global market for hydroxyurea is estimated to surpass $800 million by 2025, with a compound annual growth rate (CAGR) of approximately 4-5%, driven predominantly by the increasing burden of sickle cell disease and the expanding acceptance of hydroxyurea's benefits [2].

Key Market Drivers

Global Burden of Sickle Cell Disease

SCD affects over 300 million lives worldwide, with significant mortality burdens in sub-Saharan Africa, India, and the Middle East. The World Health Organization emphasizes hydroxyurea’s role in mitigating SCD complications, fostering its adoption in national health programs [3]. Policymaker incentives and NGO initiatives enhance hydroxyurea accessibility, boosting sales.

Evolving Clinical Guidelines and Acceptance

Clinical guidelines increasingly endorse hydroxyurea as a mainstay therapy for SCD. The National Heart, Lung, and Blood Institute (NHLBI) and other authorities recommend hydroxyurea for adult and pediatric patients, expanding its utilization scope. This consensus propels prescription volumes and supports sustained revenue streams.

Cost-Effectiveness and Affordability

Hydroxyurea’s low production costs and generic availability make it a highly cost-effective treatment option, particularly in low- and middle-income countries (LMICs). Generic formulations and licensing agreements foster wider distribution, countering market saturation concerns.

R&D and New Indications

Emerging research suggests hydroxyurea’s potential in managing other conditions, such as certain cancers and DNA repair-related disorders, possibly broadening its market scope. Companies investing in clinical trials may unlock additional revenue channels.

Market Barriers and Challenges

Regulatory and Safety Concerns

Despite its efficacy, hydroxyurea's long-term safety concerns—including risks of myelosuppression and carcinogenesis—necessitate diligent monitoring. Regulatory agencies may impose restrictions that influence prescribing behaviors.

Competitor Drugs and Therapeutic Alternatives

The rise of gene therapy and novel targeted agents, like voxelotor (Oxbryta) and crizanlizumab, offers alternative or adjunct options for SCD. These agents may erode hydroxyurea’s market share, especially where higher prices or perceived efficacy advantages are established [4].

Adherence and Dosage Optimization

Poor adherence due to side effects or lack of awareness undermines market potential. Patient education and clinician training are crucial to maximize therapeutic benefits.

Supply Chain and Access in LMICs

While low-cost generics exist, supply chain inconsistencies and regulatory hurdles in LMICs hinder widespread access. Strengthening distribution networks is essential to expanding the market.

Financial Trajectory and Investment Trends

Manufacturing and Market Penetration

Leading pharmaceutical companies like Bristol-Myers Squibb (historically) and generic manufacturers produce hydroxyurea formulations. The expansion into emerging markets sustains revenue growth. Notably, in India and Africa, government-led procurement and NGO programs drive volume sales [5].

Pricing Dynamics

In developed markets, pricing remains stable, bolstered by regulatory approvals. In LMICs, commoditization and competition among generic producers drive prices downward, sustaining affordability but constraining profit margins. The potential for value-based pricing exists in newer formulations or combination therapies.

R&D Funding and Pipeline Development

Although hydroxyurea itself is off-patent, ongoing efforts focus on optimizing dosing regimens, monitoring safety, and expanding indications. Investment in adjunct therapies or novel formulations (e.g., extended-release) could enhance the molecule’s revenue potential.

Impact of Patent Expirations and Generics

Patent expiration in the early 2000s initiated a proliferation of generic options, significantly reducing unit costs and fostering market accessibility. Continued generic competition is expected to suppress price points, favoring volume-driven revenue over margin expansion.

Future Market Outlook

The future trajectory hinges on multiple factors:

• Policy Integration and Formulary Inclusion: Increased integration of hydroxyurea into public health programs worldwide promises steady demand growth.

• Innovations in Drug Delivery: Extended-release formulations and combination therapies may improve adherence and efficacy, attracting new patient segments.

• Expansion into New Indications: Clinical trials exploring hydroxyurea in oncological and genetic disorder treatments may unlock additional revenue streams.

• Global Treatment Access Initiatives: International health agencies’ efforts to combat SCD in LMICs are poised to enhance global uptake, bolstering revenue.

• Competitive Landscape Evolution: The advent of gene editing and other advanced therapies may pose long-term threats but remain supplementary within the near-term market.

Conclusion

Hydroxyurea’s market remains robust, anchored by its proven efficacy, cost-effectiveness, and global demand driven by the sickle cell disease burden. Market growth will be sustained through expanded access in under-served regions, innovations in formulation, and potential new therapeutic indications. However, challenges such as emerging competitors, safety concerns, and regulatory hurdles necessitate adaptive strategies for market stakeholders.

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Key Takeaways

• Hydroxyurea’s market size exceeds $800 million, with steady CAGR driven by global SCD prevalence.
• Increasing acceptance and inclusion in treatment guidelines support sustained growth.
• Generic production has significantly driven affordability, expanding access in LMICs.
• Emerging therapies and gene-based treatments pose competitive threats but currently serve complementary or niche roles.
• Expansion in therapeutic indications, improved drug formulations, and global health initiatives are critical pathways for future revenue growth.

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FAQs

1. What are the primary therapeutic uses of hydroxyurea?
Hydroxyurea is primarily used to treat sickle cell disease by increasing fetal hemoglobin production and reducing vaso-occlusive crises. It also has applications in certain malignancies, including chronic myeloid leukemia and melanoma.

2. How does hydroxyurea compare economically with newer therapies?
Hydroxyurea's generic formulations make it significantly more affordable, especially in LMICs, whereas newer agents like voxelotor are costlier, limiting access in resource-poor settings.

3. What are the main barriers to hydroxyurea market expansion?
Key barriers include safety concerns, patient adherence issues, regulatory restrictions, and the emergence of alternative therapies, particularly in developed countries.

4. Will the market for hydroxyurea grow with new indications?
Potentially, yes. Ongoing research into additional uses could expand its market if safety and efficacy are confirmed in clinical trials.

5. How are global health policies influencing hydroxyurea's market?
International efforts to combat SCD through screening, awareness, and inclusion in public health programs bolster demand, especially in high-burden regions.

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References

[1] Gluckman, E., et al. (2018). Sickle cell disease: new pharmacological approaches. Nature Reviews Drug Discovery, 17(7), 451-465.

[2] Grand View Research. (2022). Hydroxyurea Market Size, Share & Trends Analysis Report.

[3] World Health Organization. (2018). Sickle Cell Disease Fact Sheet.

[4] Oyekunle, A., et al. (2020). Emerging therapies in sickle cell disease. Blood Reviews, 42, 100756.

[5] Kato, G. J., et al. (2017). Hydroxyurea for sickle cell disease. The New England Journal of Medicine, 377(4), 364-373.