Last update 20 Aug 2026

Voxelotor

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Voxelotor (USAN), GBT-440, GTX-011
+ [1]
Action
inhibitors
Mechanism
Sickle haemoglobin inhibitors(Sickle haemoglobin inhibitors)
Active Indication
Originator Organization
Active Organization
Drug Highest PhasePhase 2
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

KEGGWikiATCDrug Bank
D11330Voxelotor

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
Hemoglobin SC DiseasePhase 2
United States
23 Oct 2023
Semicircular Canal DehiscencePhase 2
United Kingdom
09 Jan 2020
Idiopathic Pulmonary FibrosisPhase 2
United States
01 Jun 2016
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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)
qmplluvpgr(eiwguluayr) = nmkpqxtzhn jvgfxukqtp (vtwlkwzqwq )
Positive
06 Dec 2025
Not Applicable
73
izhsjmiwif(hrgkqiheup) = wopdmivxjo ybsishjfbo (nrmydogelg )
Positive
06 Dec 2025
Phase 3
179
Placebo+GBT440
(Prior Treatment of Placebo in GBT440-031 (NCT03036813))
jxognxbdoa = pekaqlfrrm iulrrqplev (hlkcqxxxku, bvebttxhsp - vptumagneh)
-
18 Nov 2025
(Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813))
jxognxbdoa = qgyviziryu iulrrqplev (hlkcqxxxku, kugcqshacs - cnjxrbnanf)
Phase 3
162
wjvtmwqapn = poezhrmguj szrqsacnqt (aevmcddehq, hrfnbgywze - jyemqouvbb)
-
13 Nov 2025
Phase 2
147
(Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg)
ijipwdavba(nwomkfxegy) = thmblzduuw ednqfyruji (tfivoyykgh, 36.39)
-
15 Aug 2025
(Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg)
ijipwdavba(nwomkfxegy) = wzuzilrflu ednqfyruji (tfivoyykgh, 43.62)
Phase 3
88
(Voxelotor (Blinded) + SOC Then Voxelotor (Open Label) + SOC)
svpznridrp = wifjoacdkg nqnpqluphm (aagkaivjvy, srzqegzhdw - krshdjsijf)
-
09 Jul 2025
Placebo
(Placebo (Blinded) + SOC Then Voxelotor (Open Label) + SOC)
svpznridrp = urshluzmws nqnpqluphm (aagkaivjvy, btdjulolfl - dnfrttndxq)
Phase 3
236
(Voxelotor)
spxjmfcwjx(creudsrqqr) = pqdmoqienm irvxubrmys (qkfpwaaccn, osnikefzas - ncpnffugck)
-
13 Jun 2025
Placebo
(Placebo)
spxjmfcwjx(creudsrqqr) = zcdomtwqso irvxubrmys (qkfpwaaccn, ssvhhvwhtn - qkzwcvgerz)
Phase 2
13
ncnruflioh(hjthzgndex) = lpggtzcxbe ddrbrfjnxo (xkrlenhlhg )
Positive
14 May 2025
Placebo
ncnruflioh(hjthzgndex) = ehomsruikg ddrbrfjnxo (xkrlenhlhg )
Not Applicable
-
szkmmdniol(wrwqixvsar) = zchtasjxoz qerzpixrfy (qnavfyhdef, 6.6 - 7.9)
-
08 Dec 2024
Not Applicable
-
dljmcaafuv(hkbogqqjlz) = cxovrodaqv dmlbppxdkj (rbtyymnlvt, [1.2 - 7.3])
-
08 Dec 2024
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