Billion-Dollar Molecules: Voxelotor, A $5.4 Billion Reversible Covalent Aldehyde

Abstract. Voxelotor, a gram-daily reversible covalent aldehyde drug, is an approved first-in-class oral drug that prevents the hemoglobin polymerization that drives sickle cell disease. It was one of several agents in clinical trials acquired from Global Blood Therapeutics by Pfizer as part of an over $5 billion buyout. In this article, we dive into:

  • the mechanism of action of the first-in-class drug
  • the clinical context of the acquired portfolio
  • the drug discovery story of this new drug class

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