Why This Newly FDA Approved Sickle Cell Treatment Will Be Huge

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Global Blood Therapeutics, Inc. (NASDAQ: GBT) shares jumped on Tuesday after the firm announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older.

For some quick background, 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 impede 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.

SCD affects an estimated 100,000 people in the United States and millions of people throughout the world,  largely those whose ancestors are from sub-Saharan Africa.

Oxbryta is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. The accelerated approval is based on data from the Phase 3 Hope study. Overall, the study 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.

Ted W. Love, M.D., president and CEO of GBT, commented:

Today is a major milestone not only for GBT but, most importantly, for people living with SCD, their families and those who care for them. 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. 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.

The medicine is expected to be available through GBT’s specialty pharmacy partner network within two weeks.

Shares of GBT were last seen up about 12% at $67.58, with a 52-week range of $30.15 to $69.67. The consensus analyst price target is $93.07.

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