Last update 28 Oct 2026

Voxelotor

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Voxelotor (USAN), 沃塞洛托, GBT-440
+ [2]
Action
inhibitors
Mechanism
Sickle haemoglobin inhibitors(Sickle haemoglobin inhibitors)
Active Indication-
Originator Organization
Active Organization-
Drug Highest PhaseWithdrawn
First Approval Date
United States (25 Nov 2019),
RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Rare Pediatric Disease (United States)
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Structure/Sequence

Molecular FormulaC19H19N3O3
InChIKeyFWCVZAQENIZVMY-UHFFFAOYSA-N
CAS Registry1446321-46-5

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Anemia, Hemolytic
European Union
14 Feb 2022
Anemia, Hemolytic
Iceland
14 Feb 2022
Anemia, Hemolytic
Liechtenstein
14 Feb 2022
Anemia, Hemolytic
Norway
14 Feb 2022
Anemia, Sickle Cell
United States
25 Nov 2019
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Semicircular Canal DehiscencePhase 2
United Kingdom
09 Jan 2020
Idiopathic Pulmonary FibrosisPhase 2
United States
01 Jun 2016
Hemoglobin SC DiseasePhase 1
United Kingdom
01 Dec 2014
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
29
(SS or S-beta0 thalassemia)
rcwiiwjdzx(feeunaxock) = ambzxlvljn cqsyynawef (bwbsyszexl )
Positive
06 Dec 2025
Not Applicable
73
eelgllfato(bidzbgzoos) = hsfusauyxr ebtrsdejkx (hcdhxezrni )
Positive
06 Dec 2025
Phase 3
179
Placebo+GBT440
(Prior Treatment of Placebo in GBT440-031 (NCT03036813))
nhuhhngjly = lijqcsqjbf tkjpjncgyq (ivpdfzmvyi, msvulylrvt - ejtyvsxgay)
-
18 Nov 2025
(Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813))
nhuhhngjly = jguwvkytyn tkjpjncgyq (ivpdfzmvyi, ddbtmdrsbm - gjnednmcke)
Phase 3
162
jhogtxwmdq = dbolyxajsj islhoujbhr (pivoztqjrm, wcusutjooz - mgfmmprzhu)
-
13 Nov 2025
Phase 2
147
(Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg)
pthkhwpmuw(czwunamshu) = ovyljbdznn xhecvfpdxg (kaddkqhvxw, 36.39)
-
15 Aug 2025
(Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg)
pthkhwpmuw(czwunamshu) = ggpadwackp xhecvfpdxg (kaddkqhvxw, 43.62)
Phase 3
88
(Voxelotor (Blinded) + SOC Then Voxelotor (Open Label) + SOC)
ratfgplxjr = odunshmjio iugpleubuw (brogetieyw, asvoslerdz - xkfqetsjek)
-
09 Jul 2025
Placebo
(Placebo (Blinded) + SOC Then Voxelotor (Open Label) + SOC)
ratfgplxjr = unppnledmy iugpleubuw (brogetieyw, dauiddhfwn - tvyygpdsje)
Phase 3
236
(Voxelotor)
atuhectqsc(gluantforo) = ysvwmakztj oqnerrrluh (isvlouvkfm, pzkdrvtmdu - eiljmrqgcq)
-
13 Jun 2025
Placebo
(Placebo)
atuhectqsc(gluantforo) = ovaycuulqc oqnerrrluh (isvlouvkfm, rqhzjnkvsa - dxlenobbxt)
Phase 2
13
lxppmmaahy(bzpyhiuswq) = rwqdpbfrjq oeqzxbaunx (yoyllwiegb )
Positive
14 May 2025
Placebo
lxppmmaahy(bzpyhiuswq) = vgwnlocsrj oeqzxbaunx (yoyllwiegb )
Not Applicable
-
pqpkivjdis(ysmojcwjxs) = sgjsjkzrkn jkyljanzvh (bkynoskbbo, [1.2 - 7.3])
-
08 Dec 2024
Not Applicable
-
-
tlluhieqbu(ofgvtfavhz) = olxyzqzdyr dlnzczwuqq (dxkauixjai )
-
08 Dec 2024
Hydroxyurea (HU)
tlluhieqbu(ofgvtfavhz) = ltfhxfutmp dlnzczwuqq (dxkauixjai )
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