molecules of the year

GBT/Pfizer HbS polymerization inhibitor

FIC, oral, QD, reversible covalent HbS pol. inhibitor

accelerated FDA approval for hemolytic anemia in SCD

opt. + SBDD of natural product (5HMF) analogs

voxelotor (GBT440, OXBRYTA)

Global Blood Therapeutics, SSF, CA / Pfizer, NY

2 mins read

Voxelotor is a first-in-class, reversible covalent aldehyde drug that prevents the hemoglobin polymerization, a primary driver of sickle cell disease. Approved in 2019, this molecule represented a scientific milestone as a modern example of a reversible covalent drug being approved in a chronic indication. It is the first oral drug that improves hemoglobin levels and prevents hemolysis in sickle cell disease – in the pivotal Ph. III HOPE trial of 274 patients, more patients (51%) in the 1500-mg voxelotor group had a hemoglobin response (an increase of more than 1.0 g/dL) at 24 weeks than those in the placebo group (7%). A follow-up analysis at 72 weeks showed that improvements in hemoglobin concentrations were sustained. The molecule is of significant…

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