For people with sickle cell disease, a common, painful symptom is vaso-occlusive crisis, which is when blood circulation is blocked by sickled red blood cells. This restriction of circulation results in ischemic injury to the organ that is being supplied with the blood, and this injury brings pain. For a long time, this symptom has been something that people with sickle cell disease live with. But now the FDA has approved a drug called Adakveo, which is meant to combat this painful symptom. The acting commissioner of the FDA, Adm. Brett P. Giroir, M.D., has said that “Hope has never been higher for people living with sickle cell disease and their families and supporters.”
About Sickle Cell Disease
Sickle cell disease is a group of inherited disorders that affect the red blood cells, causing them to have an abnormal, sickle shape. This deformation of the red blood cells is caused by an abnormal protein. It is a recessive condition, meaning that both parents have to pass on the gene in order for the child to have the disease. It is most common in people of African ancestry, but it also disproportionately affects people of Hispanic descent. Healthcare professionals recommend genetic testing for couples who are aware of the disease in their family history. Symptoms include the swelling of hands and feet, symptoms of anemia such as fatigue, and jaundice. These are the earlier symptoms that usually lead doctors to a diagnosis of sickle cell disease. As the disease progresses, symptoms can include infections, delayed growth, and pain crisis. Children often only experience symptoms during pain crisis, whereas adults constantly feel the effects of the disease. Due to the misshapen red blood cells, blood circulation is restricted to different parts of the body, which can result in damage to organs such as the spleen, liver, kidneys, eyes, brain, lungs, heart, joints, skin, and bones over time. Currently the only cure for this disease is a blood and bone marrow transplant, but not all people who have sickle cell disease can receive one. Treatments exist to help with symptoms and prolong life, and healthcare professionals agree that an early diagnosis and regular medical care are extremely important.
The FDA recently approved of Adakveo, which is meant to reduce the pain crisis that people with sickle cell disease have. The reduction of this symptom can also lead to a reduction of the damage done to the organs, which greatly helps those with the disease. It is the first targeted therapy, as it inhibits selectin, which causes cells to stick together and reduce blood circulation. A clinical trial was performed in order to receive FDA approval, in which participants were given either Adakveo or a placebo. Those given Adakveo experienced less pain crisis, which also meant fewer health care visits. While the drug did reduce pain crisis, it also came with the side effects back pain, nausea, fever, and joint pain. The FDA advises that people who take Adakveo remain under monitoring by their doctor to ensure that none of these side effects become too severe. Novartis, a Swiss pharmaceutical company, was granted this approval, and the drug was also previously given Priority Review, Orphan Drug, and Breakthrough Therapy designations.
While there is only one cure for Sickle Cell Disease, which is a blood and bone marrow transplant, researchers are hopeful that the breakthrough that comes with the development of Adakveo will help to provide more treatments for this disease. The designations that this drug was given by the FDA will greatly assist in further research, and the acting commissioner has stated that there are several other current initiatives within the work being done in sickle cell disease treatment.
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