Global Blood Therapeutics, Inc. announced only a few hours ago that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta™ (previously known as Voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Oxbryta, an oral therapy taken once daily, is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. The medicine is expected to be available in the US within two weeks.
“We are proud to bring this breakthrough therapy to the SCD community. Uniquely developed from inception to treat SCD, Oxbryta embodies GBT’s commitment to develop and deliver innovative medicines for patients with overlooked, life-limiting chronic diseases. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers and advocates who have worked alongside us to develop this first-in-class therapy », said Ted W. Love, M.D., president and chief executive officer of Global Blood.
SCD affects an estimated 100,000 people in the United States and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry. SCD is a lifelong inherited blood disorder that impacts haemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. Due to a genetic mutation, people with SCD form abnormal hemoglobin known as sickle haemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which obstruct the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.
The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in hemoglobin levels, accompanied by reductions in red blood cell destruction (hemolysis). Data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization). Study of 274 patients 12 years of age and older with SCD showed that, after 24 weeks of treatment, 51.1% of patients receiving Oxbryta achieved a greater than 1 g/dL increase in hemoglobin compared with 6.5% receiving placebo (p<0.001). Results from the HOPE Study were published in June 2019 in The New England Journal of Medicine.
The med will be available within two weeks and it will bear a list price of $10,417 per month, or $125,000 per year, the company said.
More information is available at www.Oxbryta.com or 1-833-428-4968 (1-833-GBT-4YOU).
In the following video, Jonathan Sorof, MD of Global Blood Therapeutics (GBT) explains the mechanism of action of Oxbryta (Voxelotor) for SCD, as presented in the American Society of Haematology (ASH) Annual Meeting 2019: