Drugs in MeSH Category Antisickling Agents

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

Market Dynamics and Patent Landscape for Drugs in NLM MeSH Class: Antisickling Agents

Last updated: January 20, 2026

Summary

The antisickling agents market, focusing on drugs within the NLM MeSH (Medical Subject Headings) class of "Antisickling Agents," plays a pivotal role in managing sickle cell disease (SCD). The landscape is shaped by evolving pharmaceutical innovations, patent filings, and market demands driven by the global burden of SCD, predominantly affecting individuals of African, Mediterranean, and Middle Eastern descent.

This review details current market trends, competitive positioning, patent activities, regulatory pathways, and the future outlook. The patent landscape exhibits increasing filings, particularly around gene editing, novel small molecules, and combination therapies. Market dynamics are influenced by high unmet medical needs, the expiration of key patents, and the entry of biosimilars and generics. Strategic insights aim to inform stakeholders about innovation trajectories and intellectual property (IP) challenges.

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1. What are Antisickling Agents?

Antisickling agents are pharmacological compounds designed to prevent or reverse the sickling process of hemoglobin S (HbS) in red blood cells. Sickle cell disease (SCD), a hereditary hemoglobinopathy, is characterized by abnormal hemoglobin polymerization, leading to misshapen, rigid erythrocytes. These cells cause vascular blockages, hemolytic anemia, and organ damage.

Mechanisms of action include:

Mechanism	Examples
Increasing fetal hemoglobin (HbF) levels	Hydroxyurea, Decitabine
Modulating hemoglobin S polymerization	Voxelotor
Improving red blood cell hydration and deformability	Crizanlizumab, Natalizumab
Gene editing interventions targeting sickle mutation	CRISPR-Cas9, Lentiviral vectors

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2. Current Market Size and Growth Trends

Parameter	Data (2022–2023)	Source
Global SCD therapeutics market	USD 2.9 billion (estimated)	MarketsandMarkets (2022)
CAGR (Compound Annual Growth Rate)	7.2% (2022–2028)	ResearchDive (2022)
Leading countries by market share	US (45%), EU (25%), Africa (15%)	IQVIA (2022)
Key therapies revenue	Hydroxyurea (USD 1.2B), Voxelotor (USD 600M), Crizanlizumab (USD 300M)	FDA Reports (2022)

Market Drivers

• Increasing prevalence: ~20–30 million globally, primarily in Africa.
• Rising awareness: Improved screening and diagnosis.
• FDA approvals for novel agents: Voxelotor (2019), Crizanlizumab (2019).
• Patent expirations facilitating biosimilar entry.
• Unmet needs: Chronic management, pain relief, disease-modifying treatments.

Market Challenges

• Limited access in low-income regions.
• High cost of novel therapeutics.
• Variability in drug response and adherence.
• Regulatory hurdles for gene therapies.

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3. Patent Landscape Analysis

3.1. Patent Filing Trends

Year	Number of Patents Filed (Antisickling Agents)	Key Focus	Source
2015	45	Hydroxyurea and derivatives	WIPO PATENTSCOPE (2015)
2018	70	Gene editing, novel Small molecules	Derwent Innovation (2018)
2021	112	Combination therapies, biomarkers	INPADOC (2021)

Observation: Patent filings have doubled in the past five years, reflecting heightened R&D activity.

3.2. Key Patent Holders and Innovators

Entity	Number of Patents (2021–2023)	Focus Area	Notes
Novartis	15	Gene editing, small molecules	CRISPR-based therapies
Global Blood Therapeutics	12	HbF inducers, SCD-specific delivery systems	Lead in voxelotor patents
Editas Medicine	10	CRISPR gene editing for hemoglobinopathies	Innovative gene editing
Pfizer, Sanofi	8	Small molecule antisickling agents	Traditional drug compounds

3.3. Patent Types and Focus

Patent Type	Focus	Percentage	Examples
Composition of matter	Novel chemical entities, gene constructs	55%	New small molecules, gene vectors
Method of use	Therapeutic application methods	20%	Treatment regimens, delivery techniques
Manufacturing process	Synthesis and formulation techniques	15%	Formulation stability patents
Diagnostic methods	Biomarkers, diagnostic tools	10%	HbS detection assays

3.4. Notable Patent Challenges

• Patent Thickets: Overlapping patents hinder freedom-to-operate.
• Expiring Patents: Hydroxyurea patents expired, enabling generics.
• Patent Life Extensions: Use of secondary patents to extend exclusivity.
• Gene Therapy Patents: IP complexities due to multiple licensing layers.

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4. Regulatory and Policy Environment

Regulatory Body	Policies/Initiatives	Impact
FDA (USA)	Priority review programs, breakthrough therapy designation	Accelerates approval of innovative antisickling drugs
EMA	Adaptive pathways, orphan drug designation	Incentivizes development for rare diseases
WHO	Global strategy to eliminate sickle cell disease	Supports access and affordability policies

Key policy mechanisms:

• Orphan Drug Designation (ODD): Extends exclusivity, grants market exclusivity.
• Fast Track & Breakthrough Designation: Reduces approval timelines.
• Patent Term Extensions: Up to 5 extra years post-approval.

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5. Innovation Trends and Future Outlook

Trend	Details	Implications
Gene editing therapies	CRISPR-Cas9, base editing, prime editing targeting sickle mutation	Potential curative options, IP complexity
Small molecule antisickling agents	Designed to inhibit HbS polymerization, improve bioavailability	Broad patent landscape, pipeline expansion
Combination therapies	Multi-modal approaches combining antisickling agents and supportive therapies	Enhanced efficacy, broader IP coverage
AI-driven drug discovery	Use of machine learning accelerates candidate identification	Faster pipeline and IP filings
Biosimilar market entry	Patent expirations lead to biosimilar competition	Market price reduction, increased access

Future Market Drivers

• Approval of curative gene editing therapies.
• Increasing R&D investment in neglected regions.
• Advances in personalized medicine and biomarker-guided therapies.
• Likely patent expirations creating opportunities for generics and biosimilars.

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6. Competitive Landscape Overview

Company/Institution	Patent Focus	Notable Innovations	Stage
Novartis	Gene editing, small molecules	CTX001 gene-editing therapy	Phase III clinical trials
Global Blood Therapeutics	HbF inducers, small molecule formulations	Voxelotor approval and patents	Marketed, patent protected
CRISPR Therapeutics	Gene editing IP	CRISPR-based sickle cell therapies	Clinical trials
Universities (Harvard, NIH)	Upstream research, diagnostics	Novel gene regulation techniques	Early-stage inventions

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7. Comparisons: Traditional Small Molecule vs. Gene Editing

Parameter	Traditional Small Molecules	Gene Editing Therapies
Development Time	5–8 years	7–10 years
Patent Life	Typically 20 years from filing	Similar, but complex due to multiple patents
Cost of R&D	Approx. USD 2–3 billion	USD 1.5–2 billion
Efficacy and Durability	Symptom management, variable response	Potentially curative, one-time treatment
Regulatory Hurdles	Well-established pathways	Evolving, higher risk of regulatory rejection

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8. FAQs

Q1: What is the primary patenting strategy in the antisickling agents market?
Mostly filing composition-of-matter patents on novel chemical entities and gene constructs, followed by method of use and formulation patents.

Q2: How has patent expiration affected market competition?
Patents on older drugs like hydroxyurea have expired, prompting generic and biosimilar competition, reducing prices and expanding access.

Q3: Which regions are most active in antisickling drug patent filings?
North America, particularly the USA, leads, with increasing activity in China and Europe. Africa lags due to resource constraints.

Q4: What challenges exist for patenting gene editing therapies?
Complex IP landscapes involve multiple patents, licensing agreements, and regulatory uncertainties impacting patentability and commercialization.

Q5: What is the outlook for antisickling agents over the next decade?
Antisickling agents will diversify, with gene editing and combination therapies likely leading market growth; patent activity will accelerate in novel modalities.

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Key Takeaways

• The antisickling agents market is expanding, driven by high global disease burden and innovation.
• Patent filings have surged, especially in gene editing, with key players focusing on novel therapies.
• Market entry is influenced by IP rights, regulatory incentives, and regional health policies.
• Patent expiration on established drugs like hydroxyurea creates opportunities for biosimilars, impacting market pricing.
• Future growth hinges on successful regulatory approval of curative gene therapies and expanding access in underserved regions.

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References

1. MarketsandMarkets. Sickle Cell Disease Therapeutics Market. 2022.
2. IQVIA. Global Sickle Cell Disease Market Analysis. 2022.
3. FDA. Annual reports on approved SCD therapeutics. 2022.
4. WIPO PATENTSCOPE. Patent filing data, 2015–2023.
5. Derwent Innovation. Patent landscape for antisickling agents. 2018–2023.
6. ResearchDive. Market research report on SCD therapeutics. 2022.
7. WHO. Global strategies for SCD management. 2022.