IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia. The FDA had previously granted Orphan Drug Designation for IMR-687 for the treatment of patients with sickle cell disease (SCD).
IMR-687 is a highly selective and potent small molecule inhibitor of PDE9. PDE9 uniquely degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal haemoglobin, or HbF, a natural haemoglobin produced during fetal development. Increased levels of HbF in red blood cells have been demonstrated to improve symptomology and lower disease burden in patients patients with beta-thalassaemia and SCD.
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.
“We are pleased to receive this important designation from the FDA, which underscores the critical need for innovative new treatment options for patients with rare blood disorders, such as beta-thalassaemia,” said Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara. “This designation comes at an important time for our beta-thalassemia program, where we have recently initiated our Phase 2b clinical trial in the U.S. and expect to dose the first patient in the near-term. In addition, we are progressing regulatory submissions across 14 other countries, making this trial a global effort.”