Oxbryta approval a 'milestone' for sickle cell disease patients

The US Food and Drug Administration (FDA) has granted accelerated approval to Oxbryta (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older.

Manufacturer Global Blood Therapeutics (Nasdaq: GBT) has hailed Oxbryta as the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD, and the Californian company’s share price closed 11% higher on Monday.

"We now have a therapy that significantly improves hemoglobin levels, has a favorable safety profile and reduces the anemia and hemolysis"As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory trial using transcranial doppler flow velocity to demonstrate a decrease in stroke risk in children aged from two to 15 years of age. The study will be started by the end of the year.