FORMA Therapeutics, Inc. (FORMA), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for FORMA’s lead investigational agent, FT-4202, currently in clinical development as a potentially disease-modifying treatment for sickle cell disease (SCD). FT-4202 is a potent activator of pyruvate kinase-R (PKR) that, in preclinical studies, demonstrated the potential to beneficially impact both anemia and vaso-occlusive crises for people living with SCD.
“Despite newly approved therapies for SCD, a significant unmet need remains for an innovative, truly disease-modifying treatment,” said Frank Lee, chief executive officer of FORMA. “Receiving Orphan Drug designation for this investigational agent, along with the formerly granted FDA Fast Track designation and Rare Pediatric Disease designation, validates the work we are doing to address the unique struggles of patients living with the rare genetic anomaly that leads to SCD.”