Synonyms: example 8 [US20210047309] | GBT-021601 | GBT-601 | GBT021601
Compound class:
Synthetic organic
Comment: Osivelotor (GBT021601; Global Blood Therapeutics) is a clinical lead for the treatment of sickle cell disease. It stablilises sickle haemoglobin (HbS) in its oxygenated state which inhibits its polymerisation (the underlying cause of erythrocyte sickling) [3]. Based on preclinical studies osivelotor is proposed as an oral drug to reduce vaso-occlusive crises in people with sickle cell disease [1].
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No information available. |
Summary of Clinical Use |
Osivelotor (GBT021601) was progressed to phase 2/3 clinical studies. In May 2022 the FDA granted orphan drug designation which permits use of GBT021601 as a treatment for sickle cell disease [2]. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT05431088 | GBT021601-021: A Study in Adult and Pediatric Participants With SCD | Phase 2/Phase 3 Interventional | Global Blood Therapeutics | ||
NCT05632354 | GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD) | Phase 2/Phase 3 Interventional | Pfizer |