HemaQuest wins two European orphan drug designations for blood drug

HemaQuest Pharmaceuticals, a biopharmaceutical company, has received two orphan medicinal product designations from the European Commission for its product HQK-1001 for the treatment of sickle cell disease and beta thalassemia.

The new designations are in addition to orphan drug designations for both diseases granted in the US in 2008. HemaQuest has recently completed phase I clinical trials of HQK-1001, an orally administered therapeutic, in healthy subjects and plans to begin clinical studies of this compound in both sickle cell anemia and beta thalassemia in the near future.

The compound’s therapeutic potential was discovered by Susan Perrine, the company’s chief scientific officer and vice president of clinical affairs, and her colleagues at Boston University.

Ronald Berenson, president and chief executive officer of HemaQuest, said: Receiving orphan medicinal product designations for these hemoglobin disorders is critical to our overall strategy for developing HQK-1001. In contrast to the US, where beta thalassemia is relatively rare, there are several thousand patients in Europe who suffer from beta thalassemia for which there are currently no approved drugs to treat the underlying illness.

HemaQuest Pharmaceuticals is a Boston-based biopharmaceutical company focused on developing small molecule therapeutics based on its short chain fatty acid derivative (SCFAD) technologies to treat anemias and other blood disorders.