New drugs emerge to treat sickle cell disease

FDA on Monday approved the sickle cell disease drug voxelotor (Oxbryta—Global Blood Therapeutics), adding to a new wave of treatments that promise relief from the life-threatening blood disorder that largely afflicts African-Americans.

FDA on Monday approved the sickle cell disease drug voxelotor (Oxbryta—Global Blood Therapeutics), adding to a new wave of treatments that promise relief from the life-threatening blood disorder that largely afflicts African-Americans. The drug blocks a process in blood cells that can lead to anemia and organ damage, hallmarks of sickle cell disease. It is the second treatment to get FDA approval in recent weeks, after the agency approved crizanlizumab-tmca (Adakveo—Novartis) to reduce the frequency of bouts of pain that sickle cell patients can suffer. Medical officials say the new drugs have the potential to ease the complications of sickle cell disease, which afflicts approximately 100,000 Americans. More new treatments are expected. Drugmakers including Bluebird Bio, Vertex Pharmaceuticals, and Crispr Therapeutics, along with government researchers, are developing experimental therapies that may have potential to cure or provide long periods of relief. "Everybody's been waiting for this moment where the flood gate of new treatments is opening," said Biree Andemariam, MD, chief medical officer of the Sickle Cell Disease Association of America and a sickle cell specialist at UConn Health. Global Blood Therapeutics priced voxelotor at $125,000 a year, though the company said the drug would cost most health plans $96,000 a year after discounts. Novartis's drug lists for as much as $113,100 a year, depending on the weight of the patient. Drugmakers say the treatments can help cut other costs, such as hospitalization.