> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# FDA Grants Imara Pediatric Disease Status for Sickle Cell Therapy
- URL: https://www.fdaweb.com/fda-grants-imara-pediatric-disease-status-for-sickle-cell-therapy/
- Published: 2017-05-15T12:00:00.000Z
- Updated: 2026-09-14T22:23:34.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5138693

FDA has granted Imara a rare pediatric disease designation for IMR-687, the company’s lead product candidate for treating sickle cell disease. The company is conducting a [Phase 1a clinical study](http://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fclinicaltrials.gov%2Fct2%2Fshow%2FNCT02998450%3Fterm%3DIMR-687%26rank%3D1&esheet=51558411&newsitemid=20170515005466&lan=en-US&anchor=Phase+1a+clinical+study&index=3&md5=45c1f4c2005e65e1cb7a2206d385bddf&ref=fdaweb.com) to evaluate the safety and pharmacokinetics of IMR-687 in healthy volunteers. In addition, it will assess pharmacodynamic markers. Pending a positive outcome of the study this summer, Imara will initiate a Phase 2a study in adult patients with sickle cell disease later this year and then expects to initiate a Phase 2 pediatric patient study in 2018.

  
IMR-687 was designed to address the underlying pathology of sickle cell disease, according to Imara. “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,” it says. “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.”