Imara, a biotechnology company dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the company’s lead product candidate IMR-687 for the treatment of sickle cell disease.
“This designation is granted to investigational new drugs that have shown promise to address serious medical needs for patients living with rare conditions,” said James McArthur, Ph.D., Founder, President, and Chief Executive Officer of Imara. “This is an important milestone for Imara, and we look forward to continuing our efforts to advance potential new treatments for patients.”
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.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.
Filed Under: Drug Discovery
