AlDallal Salma M
Hematology Laboratory Specialist, Amiri Hospital, Kuwait City, KWT.
Cureus. 2020 Feb 26;12(2):e7105. doi: 10.7759/cureus.7105.
Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.
镰状细胞病是一种具有挑战性的血液疾病，仅在美国就影响着约10万人。目前获批的药物均无法改变该疾病的潜在病理状况。伏索利度（Voxelotor）是同类药物中的首款，是一种口服药物，可改变潜在的疾病病理状况（通过增加血红蛋白与氧气之间的亲和力）并抑制红细胞镰变。多项临床试验和病例系列记录了伏索利度治疗镰状细胞病的益处和安全性。目前，美国食品药品监督管理局已批准该药物用于治疗镰状细胞病，并授予其孤儿药地位。
