FDA approves Oxbryta to treat Sickle Cell Disease.- Global Blood Therapeutics

Global Blood Therapeutics, Inc.announced that the FDA has granted accelerated approval for Oxbryta (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Oxbryta, an oral therapy taken once daily, is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. The medicine is expected to be available through GBT�s specialty pharmacy partner network within two weeks.

SCD affects an estimated 100,000 people in the United States and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. Due to a genetic mutation, people with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled � deoxygenated, crescent-shaped and rigid. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

Comment: Novartis AG' Adakveo (crizanlizumab) was approved by the FDA on 15 November 2019, but the two drugs work differently and carry different indications. Adakveo is approved to reduce vaso-occlusive crises (VOCs) in sickle cell disease patients. Those are the terrible pain crises that can drive patients into the hospital, but for which there are limited treatment options. Oxbryta, on the other hand, was approved with a broad label for the treatment of sickle cell disease in adults and pediatric patients 12 and older. It works by inhibiting sickle hemoglobin polymerization and could be the first drug to address the underlying cause of sickle cell disease.