Case Study: USFDA Pediatric Disease Designation for Imara’s lead Product Candidate IMR-687 for Sickle Cell Disease

Imara Inc., a biotechnology company dedicated to developing novel therapeutics for patients with sickle cell disease (SCD) and other hemoglobinopathies, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to IMR-687, the company’s lead product candidate.

IMR-687 is the first SCD candidate to be designated as a drug for a rare pediatric disease, and this designation builds upon, and is complimentary to, the FDA’s earlier grant of Orphan designation.

The 21st Century Cures Act clarified the rare pediatric disease path for SCD drugs that address the pediatric manifestations of the disease. In designating IMR-687 as a drug for a rare pediatric disease, the FDA recognizes the manifestations of SCD that primarily effect children and increase pediatric morbidity, including acute splenic sequestration, cerebrovascular accidents, and aplastic anemia.

Major Reason to receive Pediatric Disease Designation

People living with sickle cell disease have limited treatment options for its devastating effects. That is why the molecule is considered for the Pediatric Disease Designation. 

Imara is conducting a Phase 1a clinical study to evaluate the safety and pharmacokinetics of IMR-687 in healthy volunteers. In addition, Imara will assess pharmacodynamic markers. Pending the positive outcome of this Phase 1 study this summer, Imara will initiate a Phase 2a study in adult patients living with sickle cell disease later this year and then expects to initiate the Phase 2 in pediatric patients in 2018.

Rare pediatric disease designation is granted to drugs that show promise to treat orphan diseases affecting fewer than 200,000 patients in the U.S., primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs. Under the FDA’s Rare Pediatric Disease Priority Review Voucher Program, a sponsor with Rare Pediatric Disease Designation who receives an approval of a new drug application (NDA) is eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application.

About IMR-687

IMR-687 was specifically designed to address the underlying 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. Pre-clinical data demonstrate IMR-687 reduces both the sickling of red blood cells and blood vessel occlusion that cause debilitating pain, organ damage, and early mortality in affected patients.