HemaQuest Pharmaceuticals announced that the US Food and Drug Administration (FDA) has awarded the company orphan drug designations for sickle cell anaemia and beta thalassemia for HQK-1001, which is an orally administered therapeutic under clinical development for these two indications.
HemaQuest president and CEO Ronald Berenson, said, "Orphan drug designations confirm the urgent medical need to develop new therapies to treat these two serious and life-threatening haemoglobin 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 anaemia and beta thalassemia."
Orphan drug status provides certain tax benefits and confers market exclusivity for a minimum of seven years after drug approval by the FDA to encourage companies to develop medicines that affect less than 200,000 people in the United States. Both haemoglobin disorders, sickle cell anaemia and beta thalassemia, fall into this category with a combined total of less than 80,000 patients suffering from these inherited blood diseases in the US.
Worldwide, sickle cell anaemia and beta thalassemia afflict millions of people. Few therapeutic alternatives exist for these serious and life-threatening diseases, which are associated with significant morbidity and reduced patient survival, creating a strong and pressing need for new treatments.
HemaQuest recently completed phase-1 clinical trials of HQK-1001 in healthy subjects and plans to begin clinical studies of this compound in both sickle cell anaemia 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.
HemaQuest Pharma, which was established in late 2007, is a biopharmaceutical company focused on developing small molecule therapeutics based on its short chain fatty acid derivative (SCFAD) technologies to treat haemoglobin diseases.