molecules of the month


oral pyruvate kinase activator

in Ph. II/III for genetic anemias

200 or 400 mg QD, follows etavopivat

J Pharmacol Exp Ther

Forma Therapeutics, Watertown, MA

chemical structure of etavopivat
3 mins read

Context. Etavopivat (Forma Therapeutics) is an oral erythrocyte pyruvate kinase activator being developed for sickle cell disease and other hemoglobinopathies, previously reported in 2019. Hydroxyurea had been the only approved drug for sickle cell disease until the “sickle cell drug boon” began in 2017 starting with approval of l-glutamine and subsequent approvals of crizanlizumab-tmca and voxelotor in 2019. Therefore, approval of the etavopivat will provide additional treatment options for the rare blood disorder. Early data from clinical trials are promising and it’s no surprise that the FDA has granted the drug Fast Track, Rare Pediatric Disease and Orphan Drug designations. Another pyruvate kinase activator, the first-in-class drug mitapivat, was approved in February for treatment of hemolytic anemia in adults with…

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