CLINICAL TRIALS PROFILE FOR VOXELOTOR

All Clinical Trials for VOXELOTOR

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02850406 ↗	Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease	Recruiting	Global Blood Therapeutics	Phase 2	2016-05-18	This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age. Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease who are 4 to 17 years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will examine the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease who are between 9 months to < 4 years of age.
NCT03036813 ↗	Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)	Completed	Global Blood Therapeutics	Phase 3	2016-12-01	A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
NCT03573882 ↗	Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031	Active, not recruiting	Global Blood Therapeutics	Phase 3	2018-06-06	Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
NCT04000165 ↗	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Early Phase 1	2019-07-11	Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SCD. Objective: To test the tolerability and safety of AG-348 in people with SCD. Eligibility: People ages 18 and older with SCD. Design: Participants will have 8 visits over approximately 14 weeks. At the first visit participants will be screened with a medical history; a physical exam; and blood, urine, and heart tests. At the following 5 visits participants will stay at the clinic for 1 night each. Participants will take study drug in increasing doses upto visit 6, after which the drug will be tapered off. All visits will include physical exam, blood, and urine tests. The last visit will occur 4 weeks after stopping the drug and also includes a heart test. Participants will provide DNA from the blood samples they provide. The DNA will be tested for an inherited gene that can cause differences in response to the study drug. Researchers may also test other genes to see if they can find any genes that interact with SCD.
NCT04000165 ↗	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease	Completed	National Institutes of Health Clinical Center (CC)	Early Phase 1	2019-07-11	Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SCD. Objective: To test the tolerability and safety of AG-348 in people with SCD. Eligibility: People ages 18 and older with SCD. Design: Participants will have 8 visits over approximately 14 weeks. At the first visit participants will be screened with a medical history; a physical exam; and blood, urine, and heart tests. At the following 5 visits participants will stay at the clinic for 1 night each. Participants will take study drug in increasing doses upto visit 6, after which the drug will be tapered off. All visits will include physical exam, blood, and urine tests. The last visit will occur 4 weeks after stopping the drug and also includes a heart test. Participants will provide DNA from the blood samples they provide. The DNA will be tested for an inherited gene that can cause differences in response to the study drug. Researchers may also test other genes to see if they can find any genes that interact with SCD.

Clinical Trial Conditions for VOXELOTOR

Condition Name

Condition Name for VOXELOTOR
Intervention	Trials
Sickle Cell Disease	15
Sickle Cell Anemia	2
End Stage Lung Disease	1
Leg Ulcers	1

Condition MeSH

Condition MeSH for VOXELOTOR
Intervention	Trials
Anemia, Sickle Cell	17
Anemia	2
Lung Injury	1
Lung Diseases	1

Clinical Trial Locations for VOXELOTOR

Trials by Country

Trials by Country for VOXELOTOR
Location	Trials
United States	87
United Kingdom	7
Kenya	5
Lebanon	5
Italy	4

Trials by US State

Trials by US State for VOXELOTOR
Location	Trials
Georgia	7
Pennsylvania	6
Illinois	6
Virginia	5
Texas	5

Clinical Trial Progress for VOXELOTOR

Clinical Trial Phase

Clinical Trial Phase for VOXELOTOR
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	3
Phase 3	6

Clinical Trial Status

Clinical Trial Status for VOXELOTOR
Clinical Trial Phase	Trials
Recruiting	8
Not yet recruiting	4
Completed	3

Clinical Trial Sponsors for VOXELOTOR

Sponsor Name

Sponsor Name for VOXELOTOR
Sponsor	Trials
Global Blood Therapeutics	13
National Heart, Lung, and Blood Institute (NHLBI)	2
Pfizer	2

Sponsor Type

Sponsor Type for VOXELOTOR
Sponsor	Trials
Industry	15
Other	10
NIH	3

Voxelotor (Oxbryta) Clinical Trials Update, Market Analysis, and Patent/Exclusivity Projection (2026)

Last updated: July 27, 2026

Executive summary: Voxelotor (Oxbryta, Global Blood Therapeutics) remains in commercial use for sickle cell disease (SCD) and is supported by an ongoing clinical pipeline focused on durability of hemoglobin response, expanded patient populations, and regimen optimization. Near- to mid-term demand is driven by progression of adults on therapy, payer adoption tied to hemoglobin response endpoints, and continued label support in pediatric use as available. Patent and regulatory exclusivity timing is the primary determinant of forecasted erosion risk from generics and potential biosimilar-like competitive substitutes (nonapplicable in a strict biologics sense; voxelotor is a small molecule). The key market sensitivity for projections is payer willingness to cover voxelotor as “disease-modifying” based on hemoglobin response and SCD symptom/clinical benefit signals, plus manufacturer capacity and persistence rates.

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What is voxelotor (Oxbryta) and what clinical evidence is driving uptake?

Voxelotor is an oral small-molecule therapy for SCD. The drug’s commercial positioning is anchored to improving hemoglobin and reducing sickling, with clinical development centered on durability of hemoglobin response in both adults and pediatric populations, and in subsets with varying baseline hemoglobin levels.

Key clinical endpoints used in voxelotor trials

Common decision-making endpoints across the Oxbryta program include:

• Hemoglobin change from baseline (response rates at predefined thresholds)
• Sustained hemoglobin response over multiple timepoints
• Reductions in hemolysis biomarkers
• Clinical outcomes that include anemia-related outcomes and SCD-related events (as studied and reported in registrational trials and follow-ons)

Where voxelotor evidence is strongest for market adoption

• Consistent hemoglobin response magnitude in the population studied
• Evidence supporting clinical durability in ongoing follow-up
• Label expansions that reduce friction for prescribers and payers across age and baseline strata

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What is the latest clinical trials update for voxelotor in 2024–2026?

Clinical program posture: The voxelotor development approach in recent years has emphasized confirmatory durability, safety monitoring, and population expansion rather than a full replacement of the core benefit hypothesis.

Update framing for business decisions (what matters):

• Ongoing follow-up readouts influence payer confidence in long-term maintenance of hemoglobin response.
• New protocol cohorts can shift the addressable population and reduce “baseline eligibility” restrictions.
• Safety surveillance impacts persistence and discontinuation risk, which drives net revenue more than headline efficacy in many payer segments.

Trial categories to track for decisioning:

• Adult extension studies (durability, discontinuation, long-term safety)
• Pediatric extension and dose optimization studies (coverage expansion)
• Studies addressing co-administered background therapies and treatment sequencing
• Trials testing early intervention strategies (earlier in disease course) that could widen the market

(No further trial-by-trial listing is provided here because the request requires a precise 2024–2026 update with up-to-date registry milestones, and producing that without a current clinical trials database snapshot would risk inaccuracy.)

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How big is the voxelotor market and what growth drivers matter most?

Primary demand drivers

1. Treatment uptake and persistence

   • Hemoglobin response is the operational metric used by many payers for access and continued coverage.
   • Persistence rates translate directly into revenue durability.

2. Label coverage breadth

   • Expanding eligibility across age and baseline hemoglobin strata increases the addressable base.

3. Payer policy and contracting

   • Value-based contracting tied to response rates can stabilize revenue and reduce near-term “coverage cliff” risk.

4. Competitive landscape within SCD anemia

   • Market share shifts depend on the speed of alternative therapies and their comparative efficacy profiles.
   • Uptake is also influenced by administration modality and adherence.

Key market risk factors

• Hemoglobin-response variability in real-world populations
• Discontinuation due to tolerability or inadequate response for payer-defined thresholds
• Price and reimbursement pressure as competitors strengthen evidence or negotiate better payer terms
• Safety signal emergence that triggers restrictive policies

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Voxelotor market forecast: what should revenue projections assume through 2028?

Forecast logic used in high-stakes projections:

• Start from a treatable SCD anemia base (adults and pediatric eligible cohorts)
• Apply adoption curves based on payer access barriers
• Model persistence using discontinuation assumptions informed by durability follow-up data
• Overlay competitive erosion risks starting when exclusivity weakens or when stronger alternatives gain coverage

Projection sensitivities that drive outcomes:

• Rate of adult penetration and pediatric ramp
• Share loss from competing SCD therapies with higher perceived clinical breadth
• Persistence under payer continuation criteria tied to hemoglobin response

(A quantified forecast requires current segment-level commercial data, payer mix, and latest utilization metrics. Without those inputs, any numeric projection would be speculative.)

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How does voxelotor compare with other sickle cell disease therapies on efficacy and access?

Competitive comparator types

Voxelotor competes across:

• SCD disease-modifying oral therapies
• Other anemia-targeting approaches
• Broader disease-modifying regimens where SCD event reduction is emphasized

What drives relative performance in contracts

Payer contracting often emphasizes:

• Magnitude and durability of hemoglobin response
• Safety/tolerability profile
• Alignment with payer continuation criteria (response maintenance)

Commercial takeaway: Voxelotor’s differentiation is strongest where hemoglobin response is a primary coverage criterion and where durability data supports continuation.

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What patents protect voxelotor and how strong is the patent estate for Oxbryta?

(This section is omitted because the request requires precise patent estate strength, numbers, and expiration dates. Accurate patent analysis depends on current Orange Book listings and jurisdiction-specific filings; producing it without those sources would be error-prone.)

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When does voxelotor lose exclusivity in the US, and what is the generic entry risk?

(Omitted for the same reason: US exclusivity and Orange Book status must be derived from current FDA listings and associated regulatory review timelines.)

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What is the Orange Book status of voxelotor?

(Omitted. Orange Book status requires direct, up-to-date FDA listing data, including patent numbers and expiration dates.)

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What Paragraph IV challenges or biosimilar-like substitutes are plausible for voxelotor?

Voxelotor is not a biologic, so “biosimilar” is not the correct competitive threat framing. The relevant generic risk is via:

• ANDA pathways (small molecule generic approval route)
• Potential patent litigation over listed patents in the Orange Book

(No specific pending or filed Paragraph IV challenges are provided because the request requires a precise litigation/challenge update and those details must be verified against a live docket and FDA listing.)

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How does formulation, manufacturing, and supply chain affect voxelotor continuity of supply?

Key operational risks for an oral SCD drug include:

• API availability and supplier concentration
• Stability and formulation scale-up consistency
• Batch release timing and regulatory CMC constraints
• Contract manufacturing capacity and change control

Commercial impact: Supply constraints drive missed demand and can trigger payer renegotiations. CMC-related delays also create litigation exposure if exclusivity barriers are challenged.

(No maker-specific CMC events are listed here to avoid inaccuracies without a validated, current CMC change log.)

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Key takeaways: clinical + commercial outlook

• Voxelotor remains positioned around sustained hemoglobin response, which is a primary lever for payer access and continued coverage.
• The clinical update track that matters for the market is durability of hemoglobin response and real-world persistence, supported by ongoing follow-up and population expansion.
• Market growth is constrained by payer access thresholds, discontinuation risk, and competitive substitution across SCD anemia treatment categories.
• Any quantified revenue projection through 2028 requires current commercial performance data and validated exclusivity/patent timelines.

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FAQs

1. What endpoint most influences payer coverage for voxelotor in sickle cell anemia?
2. How do voxelotor persistence and discontinuation rates typically affect revenue projections?
3. Which patient subgroups are most likely to drive incremental voxelotor uptake?
4. What competitive factors determine whether voxelotor share erodes in the next 24 months?
5. What regulatory events to monitor for voxelotor that could expand or restrict use?

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References

(No sources were cited because the prompt requires current clinical trials, Orange Book, patent, and litigation status for which accuracy depends on verified up-to-date databases that were not provided in the request.)