FDA Grants Rare Pediatric Disease Designation to IMR-687

Mathew Shanley
Published Online: Monday, May 15, 2017
Imara Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its lead product candidate, IMR-687.
IMR-687 is the first sickle cell disease candidate to be designated as a drug for a rare pediatric disease.

SCD is a severe, genetic form of anemia in which a mutated form of hemoglobin distorts the red blood cells at low oxygen levels into sickle, or crescent-shaped cells.
In designating IMR-687 for a rare pediatric disease, the FDA identifies the manifestations of the disorder that mainly effect children, while also increasing pediatric morbidity, including acute splenic sequestration, cerebrovascular accidents, and aplastic anemia.
Per a press release, IMR-687 was explicitly intended to address the original pathology of sickle cell disease. An orally-administered, highly potent and selective phosphodiesterase 9 (PDE9) inhibitor, IMR-687 is a potentially disease-modifying therapeutic for sickle cell disease as well as other hemoglobinopathies.
“People living with sickle cell disease have limited treatment options for its devastating effects,” said James McArthur, Ph.D., Founder, President, and CEO of Imara. “The FDA’s decision reflects its commitment to working with innovators and the patient community toward advancing safe and effective treatments for children suffering from the rare and damaging pediatric manifestations of the disease.”
“We look forward to working closely with the FDA throughout our IMR-687 clinical program,” he continued, “as this is important validation of Imara’s work in developing promising therapeutics to address serious medical needs for patients living with sickle cell disease.”
Currently, a Phase 1a clinical study is being conducted by Imara to assess the safety and pharmacokinetics of IMR-687 in healthy volunteers, and will additionally evaluate pharmacodynamic markers.
Pending a positive outcome of this study, Imara will initiate a Phase 2a study in adult patients living with sickle cell disease before the end of the year, and is expecting to initiate the Phase 2 in pediatric patients in 2018.

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