Imara Receives FDA’s Rare Pediatric Disease Designation for IMR-687 for Treatment of Sickle Cell Disease
The FDA has granted rare pediatric disease designation to drug candidate, IMR-687.
IMR-687 is the first sickle cell disease candidate to be designated as a drug for a rare pediatric disease. The selective phosphodiesterase 9 (PDE9) inhibitor, is a potentially disease-modifying therapeutic and may be effective for other hemoglobinopathies.
Imara is conducting a Phase 1a clinical study in healthy volunteers and anticipates a Phase 2a study in adult patients with sickle cell disease later this year, followed by a Phase 2 study in pediatric patients in 2018.