Voxelotor - Generic Drug Details

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

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;ORAL	213137-002	Oct 14, 2022		DISCN	Yes	No	10,806,733			Y
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-002	Oct 14, 2022		DISCN	Yes	No	11,020,382
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-001	Nov 25, 2019		DISCN	Yes	No	10,017,491		Y
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-001	Nov 25, 2019		DISCN	Yes	No	11,944,612
Global Blood Theraps	OXBRYTA	voxelotor	TABLET, FOR SUSPENSION;ORAL	216157-001	Dec 17, 2021		DISCN	Yes	No	10,017,491		Y
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;ORAL	213137-001	Nov 25, 2019		DISCN	Yes	No	11,452,720

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
Cyprus	1124097
Japan	2021113225
Croatia	P20210388
Peru	20181519	COMPUESTOS DE BENZALDEHIDO SUSTITUIDOS Y METODOS PARA SU USO EN INCREMENTAR LA OXIGENACION DEL TEJIDO
Japan	2019137699
Japan	2021113225
Eurasian Patent Organization	037091

Supplementary Protection Certificates for voxelotor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
2797416	301191	Netherlands		PRODUCT NAME: VOXELOTOR OF EEN TAUTOMEER OF FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/21/1622 20220215
2797416	22C1042	France		PRODUCT NAME: VOXELOTOR OU UN TAUTOMERE OU SEL PHARMACEUTIQUEMENT ACCEPTABLE DE CELUI-CI; REGISTRATION NO/DATE: EU/1/21/1622 20220215
2797416	CR 2022 00032	Denmark		PRODUCT NAME: VOXELOTOR ELLER EN TAUTOMER ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/21/1622 20220215
2797416	122022000052	Germany		PRODUCT NAME: VOXELOTOR ODER EIN TAUTOMER ODER PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1622 20220214
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	2022C/537	Belgium		PRODUCT NAME: VOXELOTOR OU UN TAUTOMERE OU SEL PHARMACEUTIQUEMENT ACCEPTABLE DE CELUI-CI; AUTHORISATION NUMBER AND DATE: EU/1/21/1622 20220215

Voxelotor Market Analysis and Financial Projection

Last updated: February 14, 2026

What Is Voxelotor and Its Market Position?

Voxelotor is a sickle cell disease (SCD) disease-modifying therapy developed by Global Blood Therapeutics (GBT). It is a first-in-class oral hemoglobin polymerization inhibitor approved by the US Food and Drug Administration (FDA) in November 2019 under the brand name Oxbryta. Its mechanism targets the polymerization of sickle hemoglobin, reducing sickling events and improving anemia symptoms.

Market positioning: Voxelotor's initial approval targets a niche of pediatric and adult patients with SCD. It faces competition from established therapies like hydroxyurea and emerging gene therapies, although none directly target the same molecular pathway.

What Is the Current Market Size and Demand?

The global sickle cell disease market was valued at approximately $750 million in 2022. The US market dominates this segment due to higher diagnosis and treatment rates, with an estimated 100,000 patients in the US.[1]

In the US alone, approximately 1 in 365 African-American births and 1 in 16,300 Hispanic-American births result in SCD. About 100,000 Americans have SCD, with an estimated 80% receiving some form of pharmacologic therapy.

The initial addressable market for voxelotor is roughly 75,000 patients in the US and similar figures in Europe and Africa who are eligible for disease-modifying therapy.[2]

How Is Voxelotor Performing Financially and Commercially?

Sales Performance: Since launch, voxelotor's sales have grown steadily. In 2022, GBT reported $146 million in worldwide revenue, up from $55 million in 2020.[3] US sales were approximately $100 million, reflecting market penetration.

Pricing Strategy: Voxelotor’s wholesale acquisition cost (WAC) is approximately $50,000 annually per patient. Payers negotiate discounts, with net prices estimated around $35,000–$40,000 per year. The high cost limits access in some cases, especially outside the US.

Market Access: Insurance reimbursement covers most patients, though prior authorizations and restrictive formularies delay access. Post-approval expansion into pediatric populations (ages 4 and above) increased eligible patient numbers and improved sales prospects.

Physician Adoption: Growing clinical awareness has led to increased prescriptions, but uptake remains constrained by clinician familiarity and competition from hydroxyurea, which remains the first-line treatment.

What Are Key Competitive and Regulatory Factors?

Competition: Hydroxyurea has been the standard before voxelotor, but it has off-target toxicities and adherence issues. The emergence of gene therapies, such as CRISPR-Cas9-based interventions, could disrupt the market but are still in early development stages.

Regulatory Landscape: Beyond the US, approval has been granted in the European Union (EMA) in May 2021. Many African nations face barriers due to healthcare infrastructure limitations and regulatory delays, restricting market expansion.

Pipeline and Expansion: GBT submitted a supplemental Biologics License Application (sBLA) for pediatric use; approval could expand the market. GBT also explores concomitant use with other SCD therapies, potentially increasing treatment complexity and revenue.

What Are the Market Growth Drivers and Challenges?

Drivers:

• Growing awareness and diagnosis of SCD globally.
• Increasing insurance coverage and payer support in the US and Europe.
• Evolving clinical guidelines incorporating voxelotor as a recommended therapeutic option.
• Rising demand for oral, convenient therapies compared to injectable alternatives.

Challenges:

• Cost concerns limit access in lower-income markets.
• Competition from alternative treatments, especially gene therapies anticipated to provide one-time cures.
• Limited long-term safety and efficacy data as a relatively new drug.
• Variability in regulatory and healthcare infrastructure worldwide.

What Are Future Financial Trajectories and Market Opportunities?

Forecasts: Industry analysts project the global sickle cell disease drug market will reach approximately $1.1 billion by 2025, with voxelotor capturing a significant share due to its approved status and growing patient base.[4] The compound annual growth rate (CAGR) is expected to be 8-10%.

Expansion Potential: Market expansion depends on:

• Approval for pediatric use, widening eligibility.
• Entry into underserved markets, including Africa and Asia.
• Development of combination therapies to improve efficacy.
• Inclusion in broader disease management guidelines.

Risks: Price sensitivity, regulatory delays, and emergence of superior therapies could suppress revenue growth.

Key Takeaways

• Voxelotor, approved in 2019, addresses a niche in SCD management but is gaining market share gradually.
• US sales reached approximately $100 million in 2022, with steady growth.
• The high treatment cost poses access challenges, especially outside the US.
• Competition from hydroxyurea and future gene therapies remains significant.
• Global expansion hinges on regulatory approvals, cost management, and healthcare infrastructure development.
• The market's future largely depends on clinical guideline integration, payer policies, and innovation rates in therapeutics.

FAQs

1. What is voxelotor’s primary mechanism of action?
It inhibits sickle hemoglobin polymerization, reducing sickling and anemia in SCD patients.

2. How does voxelotor compare to hydroxyurea?
Voxelotor provides a direct mechanism targeting hemoglobin polymerization, whereas hydroxyurea increases fetal hemoglobin and has more toxicity concerns and adherence issues.

3. Are there new pipeline therapies for SCD?
Yes, gene-editing approaches like CRISPR-based therapies are in clinical trials, offering potential curative options but are not yet commercially available.

4. What are barriers to voxelotor’s global adoption?
Regulatory delays, healthcare infrastructure limits, high treatment costs, and competition from emerging therapies.

5. What is the outlook for voxelotor’s revenue growth?
Stable growth is projected through 2025, driven by market expansion, pediatric approvals, and potential combination treatments, though high costs and competition pose challenges.

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Sources
[1] Grand View Research, "Sickle Cell Disease Market," 2022.
[2] GBT Investor Presentation, 2022.
[3] GBT Quarterly Financial Statements, 2022.
[4] EvaluatePharma, 2022.