The Food and Drug Administration (FDA) has granted Orphan Drug designation to FT-4202 (FORMA Therapeutics) for the treatment of sickle cell disease (SCD).

FT-4202 is a novel, small molecule allosteric activator of erythrocyte pyruvate kinase-R (PKR). Through PKR activation, FT-4202 is expected to decrease 2,3-DPG, reducing sickle hemoglobin polymerization and red blood cell sickling; and increase ATP, promoting red blood cell repair and reducing hemolysis. 

The Orphan Drug designation is supported by preclinical studies that demonstrated the potential benefit of FT-4202 to increase hemoglobin levels and decrease vaso-occlusive crises in SCD. The Company is currently enrolling patients with SCD in a phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics of FT-4202.

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The FDA previously granted Fast Track and Rare Pediatric Disease designation to FT-4202 for this indication.

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This article originally appeared on MPR