IMARA Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of haemoglobin, announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric Disease designation for its lead clinical asset, IMR-687, for the treatment of β-thalassaemia.
The FDA had previously granted Orphan Drug designation for IMR-687 for the treatment of patients with beta-thalassemia and Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of patients with sickle cell disease.
“Fast Track designation helps us create the opportunity to potentially accelerate the development of IMR-687 in β-thalassemia. Rare Pediatric designation reflects the agency’s recognition that β-thalassaemia is a serious disease, with symptoms that manifest in childhood and progress over time into adulthood,” said Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara.
Τhe company recently launched a Phase 2b clinical trial of IMR-687 in adult patients with β-thalassaemia and expects to dose the first patient in the near-term. Regulatory submissions are underway in 14 countries and screening has initiated for the Phase 2b clinical trial.
About Fast Track Designation
The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It also enables eligibility for Accelerated Approval and Priority Review, as well as a rolling review of a company’s New Drug Application, if relevant criteria are met.
About Rare Pediatric Disease Designation
Under the FDA’s Rare Pediatric Disease (RPD) program, a sponsor who receives marketing approval for a product with an RPD designation may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application. The FDA defines a „rare pediatric disease“ as a rare disease that affects fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals from age zero to 18.
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. Lower levels of cGMP are often found in people with sickle cell disease and β-thalassaemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation.
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 with sickle cell disease and patients with β-thalassaemia.
Read more information about IMR-687 & PDE9 HERE
Read the Imara Inc. Full Press Release HERE