HemaQuest gets orphan-drug designations

HemaQuest Pharmaceuticals Inc., a year-old Boston company focused on developing new treatments for blood diseases, said today that its HQK-1001 drug candidate has won orphan drug status from the US Food and Drug Administration

HQK-1001 has been granted orphan drug designations for sickle cell anemia and beta thalassemia, the company said in a press release.

The US Orphan Drug Act rewards companies that develop drugs for rare diseases by giving them seven years to sell each new treatment without competition in the United States from other manufacturers.

According to HemaQuest, sickle cell anemia and beta thalassemia are inherited blood diseases that affect fewer than 80,000 patients in the United States.

HemaQuest president and chief executive Ronald Berenson said in a statement, "Orphan drug designations confirm the urgent medical need to develop new therapies to treat these two serious and life-threatening hemoglobin disorders. These designations by the FDA also provide us with strong incentives for our novel proprietary drug candidate, HQK-1001, which initially is being developed to treat both sickle cell anemia and beta thalassemia."

HemaQuest said it recently completed early clinical trials of HQK-1001 in healthy subjects and plans to begin clinical studies of this compound in both sickle cell anemia and beta thalassemia in the near future.

Research at Boston University was key in identifying HQK-1001's potential for treating blood diseases, the company said.
(By Chris Reidy, Globe staff)