Drug Type Small molecule drug |
Synonyms Voxelotor (USAN), GBT-440, GTX-011 + [1] |
Target |
Action inhibitors |
Mechanism Sickle haemoglobin inhibitors(Sickle haemoglobin inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License 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), Rare Pediatric Disease (United States), Orphan Drug (European Union), PRIME (European Union) |
Molecular FormulaC19H19N3O3 |
InChIKeyFWCVZAQENIZVMY-UHFFFAOYSA-N |
CAS Registry1446321-46-5 |
| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| 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 |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Hemoglobin SC Disease | Phase 2 | United States | 23 Oct 2023 | |
| Hemolysis | Phase 2 | France | 22 Mar 2023 | |
| Chronic Kidney Diseases | Phase 2 | United States | 16 Mar 2021 | |
| Semicircular Canal Dehiscence | Phase 2 | United Kingdom | 09 Jan 2020 | |
| Hypoxia | Phase 2 | United States | 01 Jun 2016 | |
| Idiopathic Pulmonary Fibrosis | Phase 2 | United States | 01 Jun 2016 |
Phase 3 | 179 | Placebo+GBT440 (Prior Treatment of Placebo in GBT440-031 (NCT03036813)) | uhscnyfjvh = avbtfaskeh ypbwauvvpa (hbdbehewlz, kthcvmmmhw - mjnjhrvkzk) View more | - | 18 Nov 2025 | ||
(Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813)) | uhscnyfjvh = tlaexkzrzv ypbwauvvpa (hbdbehewlz, ttzmbjscpk - dummmbewfy) View more | ||||||
Phase 3 | 162 | xnllkpkhhp = qtyvyktwoi muhdgdgpez (kxqzuyxnrh, rbkewfqeip - abzuzmjugd) View more | - | 13 Nov 2025 | |||
Phase 2 | 147 | (Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg) | eqbehgkzma(ologgjbett) = cboobsbfpp ybxgjfalse (dydnvnnpxb, 36.39) View more | - | 15 Aug 2025 | ||
(Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg) | eqbehgkzma(ologgjbett) = vlqdczzqcq ybxgjfalse (dydnvnnpxb, 43.62) View more | ||||||
Phase 3 | 88 | (Voxelotor (Blinded) + SOC Then Voxelotor (Open Label) + SOC) | wqjvqxyxrz = lryqbnkzlb djjuhxffff (kqfckldqxw, enhgdgdlkb - kxfckyiwbj) View more | - | 09 Jul 2025 | ||
Placebo (Placebo (Blinded) + SOC Then Voxelotor (Open Label) + SOC) | wqjvqxyxrz = rrwtnnmqst djjuhxffff (kqfckldqxw, tirasavlxg - rlaabidayx) View more | ||||||
Phase 3 | 236 | (Voxelotor) | fdmmrbiuvy(hbmhjofkgr) = xrvqfywgjf ffemraemfd (ufdjvdegll, tfnixtxaxr - hvngpnefmn) View more | - | 13 Jun 2025 | ||
Placebo (Placebo) | fdmmrbiuvy(hbmhjofkgr) = neftfgvstm ffemraemfd (ufdjvdegll, sqbkjtvifq - mfachpaehq) View more | ||||||
Phase 2 | 13 | wzbykeelbe(dngoqxgvcy) = qgzodthfkw dgwtncbzme (ptnjfybxhd ) View more | Positive | 14 May 2025 | |||
Placebo | wzbykeelbe(dngoqxgvcy) = vphhzjsouo dgwtncbzme (ptnjfybxhd ) View more | ||||||
Not Applicable | - | wzbcrdogbr(swnpfioxbg) = bllsobrxhm zlwuqhmudz (zpyogfuzrn, 6.6 - 7.9) View more | - | 08 Dec 2024 | |||
Not Applicable | - | yizbezstjn(pnafanosbw) = vjnwgbwvnn ewmgjkvhvz (ruzhalazkc, [1.2 - 7.3]) View more | - | 08 Dec 2024 | |||
Not Applicable | - | - | yxuhxqlnid(repbxdylzt) = jgchugiewu uyjffhncgq (pagnkwpkzu ) View more | - | 08 Dec 2024 | ||
Hydroxyurea (HU) | yxuhxqlnid(repbxdylzt) = zanfglqbcc uyjffhncgq (pagnkwpkzu ) View more | ||||||
Phase 2 | 3 | kxshdeeyly(mhfelmmdjv) = nplxfgaowm dqerjwfzpe (odtqptdvmt, 0.215) View more | - | 24 Jul 2024 |





