FDA Grants Rare Pediatric Disease Designation to New Sickle Cell Treatment Candidate

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to IMR-687 for patients with sickle cell disease (SCD). IMR-687 is an orally administered phosphodiesterase 9 inhibitor that aims to reduce the sickling of red blood cells and blood vessel occlusion.

This is the first designation of its kind for an SCD treatment. Rare Pediatric Disease designation is granted to drugs that treat diseases affecting fewer than 200,000 patients in the United States. These agents will now have access to the FDA’s expedited review and approval programs.

The FDA previously granted orphan designation to IMR-687, which is being evaluated in a phase Ia trial of healthy volunteers. If IMR-687 is shown to be safe in this population, the drug’s manufacturer plans to initiate a phase IIa study in adult patients with SCD later this year.

Source: Imara press release, May 15, 2017.