- As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial doppler (TCD) flow velocity to demonstrate a decrease in stroke risk in children 2 to 15 years of age. The study will be initiated by the end of the year.
The biotech company confirmed the approval late Monday. The drug, called Oxbryta, is a daily tablet to treat sickle cell disease in patients ages 12 and older. It's the first drug that targets sickle hemoglobin polymerization, the root cause of sickle cell disease.
"Today is a major milestone not only for GBT but, more importantly, for people living with SCD, their families and those who care for them," Chief Executive Ted Love said in a written statement. "When we started our journey with the SCD community more than eight years ago, we set out to transform the way this devastating, lifelong disease is treated."
Sickle cell disease affects an estimated 100,000 people in the U.S. and millions worldwide, the biotech company said in a news release.
The inherited disorder impacts hemoglobin, a protein carried by red blood cells to deliver oxygen to tissues and organs. A genetic mutation causes patients to generate abnormal hemoglobin. A process called hemoglobin polymerization causes the red blood cells to become sickled.
FDA officials approved Oxbryta in sickle cell treatment three months earlier than expected based on a Phase 3 study called Hope. After 24 weeks of treatment, 51.1% of patients who took Oxbryta showed a hemoglobin increase vs. 6.5% of those who took the placebo.
