Imara Receives FDA’s Rare Pediatric Disease Designation for IMR-687 for Treatment of Sickle Cell Disease

May 17, 2017

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.

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