Global Blood Therapeutics wins first FDA approval with sickle cell disease med

Nov. 26, 2019

The U.S. Food and Drug Administration has approved a drug from Global Blood Therapeutics to treat sickle cell disease in adults and children 12 years or older. The drug, Oxbryta, was granted a Fast Track designation as well as an Orphan Drug designation.

Oxbryta will be priced at $10,417 per month and will become the first to target the underlying cause of the disease rather than symptoms.

The drugmaker says Oxbryta can be used in combination with Novartis’s drug, Adakveo, which nabbed FDA approval just last week.

"Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” said acting FDA commissioner Brett Giroir. "Our scientific investments have brought us to a point where we have many more tools available in the battle against sickle cell disease, which presents daily challenges for those living with it. We remain committed to raising the profile of this disease as a public health priority and to approving new therapies that are proven to be safe and effective. Together with improved provider education, patient empowerment, and improved care delivery systems, these newly approved drugs have the potential to immediately impact people living with SCD."

Nonclinical studies have demonstrated that Oxbryta inhibits red blood cell sickling, improves red blood cell deformability (ability of a red blood cell to change shape) and improves the blood’s ability to flow.

According to the FDA, Oxbryta’s approval was based on the results of a clinical trial with 274 patients with sickle cell disease. In the study, 90 patients received 1500 mg of Oxbryta, 92 patients received 900 mg of Oxbryta and 92 patients received a placebo. Effectiveness was based on an increase in hemoglobin response rate in patients who received 1500 mg of Oxbryta, which was 51.1% for these patients compared to 6.5% in the placebo group.

Read the full FDA release