On Monday, the U.S. Food and Drug Administration (FDA) announced accelerated approval of Oxbryta (voxelotor) for the treatment of sickle cell disease in patients aged 12 and above.
FDA approval was granted to Global Blood Therapeutics, the manufacturer of Oxbryta.
The company said in a press release, “Oxbryta is the first approved treatment for sickle cell disease that directly inhibits the root cause of the disease, sickle hemoglobin polymerization.”
Non-clinical studies of the drug have shown that it inhibits red blood cell (RBC) sickling and improves its deformability, enhancing overall blood flow.
The FDA said the recommended Oxbryta dosage is 1,500 mg orally, which should be taken once a day with or without food. The recommended dosage is 1,000 mg orally once a day daily for patients with severe liver damage.
Human trials were conducted on 274 patients with sickle cell disease, of which, 90 patients were given 1,500 mg Oxbryta, 92 patients were given 900 mg, while the remaining 92 patients were given a placebo.
After 24 weeks, more than 51 percent of patients who received Oxbryta 1,500 mg had a greater hemoglobin response when compared with only 6.5 percent of patients who were given a placebo.
Some of the common side effects of the drug include a headache, abdominal pain, diarrhea, fatigue, nausea, fever, and rash. Oxbryta is set to hit the market with a couple of weeks, which will be available through a specialty pharmacy chain.
