FDA grants orphan drug designation to sickle cell treatment

Release Date: 12-Feb-2017

Imara therapeutics, a leading biopharmaceutical announced that US Food and Drug Administration have granted orphan drug designation to its novel drug, IMR-657 to treat patients with Sickle cell disease. Presently, IMR-657 is stepping towards its first clinical phase to investigate its safety and pharmo-kinetics in patients with sickle cell. The study is currently being examined over patients between the ages of 18-55 years having sickle cell.

IMR-687 is an orally-administered selective phosphodiesterase 9 (PDE9) inhibitor. Pre-clinical data has demonstrated that IMR-687 reduces both the sickling of red blood cells and blood vessel obstruction that cause sapping pain, organ damage, and early mortality in affected sickle cell patients. Sickle cell is a rare genetic disorder causing hard, sticky and C-shaped red blood cells which further blocks smaller cell resulting into pain as well as increases the risk of infection, acute chest syndrome and stroke.

FDA's Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

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