Voxelotor receives FDA approval for sickle cell disease therapyDecember 2, 2019
The US FDA has announced approval for the innovative drug voxelotor (Oxbryta) for the treatment of sickle cell anemia in adults and children of 12 years and above.
The drug developed by Global Blood Therapeutics will be priced at $10,417 per month or around $125,000 per year, reports Reuters.
Voxelotor works by preventing the sickling of red blood cells which is the main cause of the disease. The sickled RBCs restrict the flow of oxygen in blood vessels and can entangle resulting in blood clots. The blood clots may lead to severe pain in the back, chest, hands and feet and cause organ damage. Reducing the deformation of RBCs can thus significantly relieve symptoms in sickle cell patients.
Global Blood Therapeutics will be releasing the results from a long term confirmatory trial of the drug by tracking the symptom reduction in the patients, by the end of 2019.
Voxelotor will be used in combination with Novartis’s drug, Adakveo, in sickle cell patients suffering from frequent pain crises, noted the company.
Novartis AG’s Adakveo had recently won US regulatory clearance in reducing the incidence of sickle cell-related pain, which is a common and debilitating symptom of the disease.
Sickle cell disease is an inherited disorder of haemoglobin. It is most prevalent in sub-Saharan African countries. According to WHO, over 300,000 children are born worldwide with severe forms of the disease, the majority in low and middle income countries.