VOXELOTOR - Generic Drug Details

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

Voxelotor is the generic ingredient in one branded drug marketed by Global Blood Theraps and is included in two NDAs. There are eleven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Voxelotor has two hundred and ninety-seven patent family members in forty-two countries.

One supplier is listed for this compound.

Summary for VOXELOTOR

International Patents:	297
US Patents:	11
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	48
Clinical Trials:	18
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for VOXELOTOR
What excipients (inactive ingredients) are in VOXELOTOR?	VOXELOTOR excipients list
DailyMed Link:	VOXELOTOR at DailyMed

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

Constraining patent/regulatory exclusivity:

10,493,035

Dosage:

TABLET;ORAL

Constraining patent/regulatory exclusivity:

9,447,071

Dosage:

TABLET, FOR SUSPENSION;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 VOXELOTOR

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

Sponsor	Phase
Inova Health Care Services	PHASE2
Pfizer	PHASE2
Pfizer	Phase 1

See all VOXELOTOR clinical trials

Pharmacology for VOXELOTOR

Drug Class	Hemoglobin S Polymerization Inhibitor
Mechanism of Action	Cytochrome P450 3A4 Inhibitors Hemoglobin S Polymerization Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for VOXELOTOR

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

Paragraph IV (Patent) Challenges for VOXELOTOR

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
OXBRYTA	Tablets	voxelotor	300 mg and 500 mg	213137	2	2023-11-27
OXBRYTA	Tablets for Oral Suspension	voxelotor	300 mg	216157	1	2023-11-27

US Patents and Regulatory Information for VOXELOTOR

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Global Blood Theraps	OXBRYTA	voxelotor	TABLET, FOR SUSPENSION;ORAL	216157-001	Dec 17, 2021		DISCN	Yes	No	10,806,733			Y
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-002	Oct 14, 2022		DISCN	Yes	No	9,248,199
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-001	Nov 25, 2019		DISCN	Yes	No	10,493,035		Y
Global Blood Theraps	OXBRYTA	voxelotor	TABLET, FOR SUSPENSION;ORAL	216157-001	Dec 17, 2021		DISCN	Yes	No

EU/EMA Drug Approvals for VOXELOTOR

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Pfizer Europe MA EEIG	Oxbryta	Voxelotor	EMEA/H/C/004869Oxbryta is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.	Authorised	no	no	yes	2022-02-14

International Patents for VOXELOTOR

Country	Patent Number	Title	Estimated Expiration
Guatemala	201400136	COMPUESTOS Y MÉTODOS DE BENZALDEHÍDO SUBSTITUÍDO PARA SU USO EN INCREMENTAR LA OXIGENACIÓN DE LOS TEJIDOS
Peru	20221914
South Africa	202401707	CRYSTALLINE POLYMORPHS OF THE FREE BASE OF 2-HYDROXY-6-((2-(1-ISOPROPYL-1H-PYRAZOL-5-YL)PYRIDIN-3-YL)METHOXY)BENZALDEHYDE
Spain	2860648

Supplementary Protection Certificates for VOXELOTOR

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2797416	SPC/GB22/039	United Kingdom		PRODUCT NAME: VOXELOTOR OR PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REGISTERED: UK EU/1/21/1622(FOR NI) 20220215; UK FURTHER MA ON IPSUM 20220215
2797416	PA2022517	Lithuania		PRODUCT NAME: VOKSELOTORAS ARBA TAUTOMERAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA ; REGISTRATION NO/DATE: EU/1/21/1622 20220214
2797416	122022000052	Germany		PRODUCT NAME: VOXELOTOR ODER EIN TAUTOMER ODER PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1622 20220214
2797416	C202230036	Spain		PRODUCT NAME: VOXELOTOR O UN TAUTOMERO O SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO; NATIONAL AUTHORISATION NUMBER: EU/1/21/1622; DATE OF AUTHORISATION: 20220214; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/21/1622; DATE OF FIRST AUTHORISATION IN EEA: 20220214

Market Dynamics and Financial Trajectory for Voxelotor

Last updated: July 29, 2025

Introduction

Voxelotor, marketed under the brand name Oxbryta, is a groundbreaking oral therapy developed by Global Blood Therapeutics (GBT) for the treatment of sickle cell disease (SCD). Approved by the U.S. Food and Drug Administration (FDA) in November 2019, voxelotor represents a novel mechanism targeting hemoglobin polymerization, a hallmark of SCD. Its market trajectory is influenced by evolving clinical data, regulatory landscapes, competitive pressures, and broader healthcare trends. This analysis explores these market dynamics and forecasts voxelotor’s financial trajectory over the coming years.

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Market Landscape for Sickle Cell Disease Treatments

Sickle cell disease affects approximately 100,000 individuals in the United States and millions globally, primarily within African, Mediterranean, Middle Eastern, and Indian populations[1]. Historically, therapeutic options for SCD have been limited to supportive care, hydroxyurea, and hematopoietic stem cell transplantation. The unmet medical need remains substantial, providing a fertile environment for innovative therapies like voxelotor.

The global SCD treatments market was valued at around $1.2 billion in 2021 and is projected to grow at a CAGR of 8-10% over the next five years[2]. The mission to develop disease-modifying therapies is central to pharmaceutical strategies for this under-served population.

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Mechanism of Action and Clinical Evidence

Voxelotor functions as a hemoglobin S polymerization inhibitor, increasing hemoglobin levels and reducing hemolytic anemia. Clinical trials, notably the Phase III HOPE Study, demonstrated significant increases in hemoglobin and improvements in markers of hemolysis, establishing efficacy and favorable safety profiles[3].

Key clinical data features:

• Increases in hemoglobin levels by approximately 1 g/dL compared to placebo.
• Reduction in hemolysis markers, such as indirect bilirubin.
• Well-tolerance with manageable side effects.

This pharmacodynamic profile signals a shift towards disease-modifying therapy, potentially altering the standard of care in SCD and expanding patient access.

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Regulatory and Commercial Positioning

Following FDA approval, voxelotor secured approvals in other jurisdictions, including the European Union and Canada, expanding its global footprint[4]. GBT’s strategic focus involves integrating voxelotor into existing care paradigms, collaborating with healthcare providers to expand access, and advocating for broader insurance coverage.

The commercial success hinges on:

• Pricing strategies aligning with value-based care models.
• Reimbursement frameworks that facilitate patient access.
• Physician and patient education to promote early adoption.

As of 2022, GBT has set the wholesale acquisition cost at approximately $54,000 per year per patient[5], positioning voxelotor as a premium therapy with a targeted market.

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Market Challenges and Competitive Landscape

While voxelotor is pioneering, it faces several challenges:

• Pricing pressures: The high cost may limit access, especially in low- and middle-income countries where SCD prevalence is high.
• Competition from emerging therapies: Gene editing approaches (e.g., CRISPR-based therapies) and other novel modalities are advancing rapidly.
• Adherence issues: Chronic oral therapy demands consistent patient adherence.
• Limited data in pediatric populations: While ongoing trials aim to expand indications, initial data are predominantly in adults.

Major competitors include traditional therapies like hydroxyurea and emerging drug candidates such as voxelotor’s potential rivals in hemoglobin modulation.

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Financial Trajectory and Revenue Forecast

In 2021, GBT reported voxelotor revenues of approximately $133 million, reflecting initial adoption in the US[6]. Post-approval, sales growth has been driven by expanding indications and increasing healthcare provider familiarity.

Projected revenue streams:

• 2023-2025: Continued adoption driven by increased awareness and expanded payer coverage. GBT projects revenues potentially reaching $300-$400 million annually by 2025, contingent upon market penetration and competitive dynamics[7].
• Market penetration: Estimated at 15-20% of the adult SCD patient population within five years, considering diagnosis rates, healthcare access, and provider acceptance.

Cost management strategies, investments in patient support programs, and competitive positioning will influence profitability and cash flows. The company’s pipeline, including next-generation hemoglobin modulators, could substitute or supplement voxelotor, affecting future sales.

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Regulatory and Policy Impacts

Policy initiatives emphasizing rare disease therapies and orphan drug incentives support voxelotor's market expansion. Orphan drug designation provides benefits like extended exclusivity, which can delay generic competition and secure premium pricing.

The evolving landscape of healthcare regulations, reimbursement practices, and international approvals will further shape financial prospects.

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Emerging Opportunities and Risks

Opportunities:

• Pediatric approvals: Once approved, expanding indications can exponentially grow the patient base.
• Combination therapies: Synergistic use with other SCD therapies could optimize outcomes.
• Global health initiatives: Collaborations with NGOs and government agencies can facilitate access in high-berting regions.

Risks:

• Pricing and payor constraints may impair revenue growth.
• Competitive innovations could erode market share.
• Long-term safety data remains critical; any adverse findings may impact adoption.

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Conclusion and Strategic Outlook

Voxelotor’s market dynamics reflect a confluence of clinical validation, regulatory momentum, and healthcare needs for SCD. While current revenue streams demonstrate promising growth, future success depends on widening access, reducing costs, and demonstrating long-term benefits.

GBTs’ strategic focus on expanding indications, increasing global approvals, and managing competitive threats will determine its financial trajectory. With robust unmet needs and favorable policy environments, voxelotor has substantial potential to become a mainstay in SCD management, provided it navigates market challenges effectively.

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Key Takeaways

• Market growth potential: The global SCD therapeutics market is expanding, driven by the need for disease-modifying drugs like voxelotor.
• Revenue outlook: Anticipated to reach $300-$400 million annually by 2025, contingent upon expanded indications and payer coverage.
• Competitive landscape: While pioneering, voxelotor faces emerging gene therapies and pricing pressures, necessitating strategic positioning.
• Regulatory support: Orphan drug designation and international approvals bolster market exclusivity and pricing power.
• Strategic priorities: Expanding access, demonstrating long-term efficacy, and exploring combination therapies are critical to long-term financial success.

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FAQs

1. What is the primary mechanism of voxelotor in treating sickle cell disease?
Voxelotor inhibits hemoglobin S polymerization, increasing hemoglobin's oxygen affinity and reducing sickling and hemolysis.

2. How does voxelotor’s market penetration compare with other SCD therapies?
As a recent market entrant, voxelotor’s adoption is growing but remains behind established supportive therapies like hydroxyurea. Its innovative mechanism positions it for rapid uptake where approved.

3. What factors could limit voxelotor’s revenue growth?
High costs, limited pediatric data, competition from gene therapies, and reimbursement barriers could suppress revenue expansion.

4. How do regulatory approvals influence voxelotor’s fiscal prospects?
Regulatory clearances, especially in key markets like the EU and Canada, expand sales opportunities and justify premium pricing strategies.

5. What future developments could impact voxelotor’s financial trajectory?
Indications expansion, combination therapy trials, and global health initiatives could enhance revenues, while challenges like market competition and safety concerns pose risks.

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References:

[1] National Institutes of Health. Sickle Cell Disease Fact Sheet.
[2] MarketsandMarkets. Sickle Cell Disease Therapeutics Market Forecast. 2022.
[3] Ataga et al., New England Journal of Medicine, 2019.
[4] European Medicines Agency. Judicial approval for Oxbryta.
[5] Global Blood Therapeutics. Pricing and Reimbursement Strategy, 2022.
[6] GBT Form 10-K, 2021.
[7] GBT Investor Presentation, 2022.