While bad news about COVID-19 dominates the headlines, we have better news for sickle cell patients from two biotechs providing much-needed hope in the face of longstanding disparities.
The context: Sickle cell disease is an inherited, lifelong disease that can cause extreme pain, organ damage, and infection, among other serious complications. It affects approximately 100K people in the U.S.—the vast majority of whom are Black, African American, or Hispanic.
But sickle cell patients have new reasons for hope—as Global Blood Therapeutics (GBT) and bluebird bio (both BIO members) have good news to report.
The FDA granted accelerated approval to GBT’s pioneering Oxbryta for use in children ages 4-12, extending the previous application approved for ages 12+, the company announced Friday. The FDA also approved a grape-flavored suspension that’s easier to take.
“Due to the many medical complications associated with sickle cell disease, kids face challenges every day in their ability to go to school and have a typical childhood,” said Dr. Lewis Hsu, Chief Medical Officer of the Sickle Cell Disease Association of America. “Adding another new FDA-approved treatment option for children will enhance care and provide more choices for children living with sickle cell disease.”
GBT also announced promising data from a phase 1 trial of a one-daily pill, which “has the potential to improve clinical outcomes in people living with SCD, while reducing pill burden,” said the study’s lead investigator Dr. Clark Brown.
bluebird bio, meanwhile, announced updated results from the phase 1/2 trial of a gene therapy: “In addition to continued complete resolution of severe vaso-occlusive events (VOEs), patients in Group C achieved near normal levels of key hemolysis markers and experienced sustained improvements in patient-reported quality of life following treatment,” says the company’s statement.
The therapy “has the potential to improve the day-to-day reality of people living with sickle cell disease by eliminating the disruptive, painful crises that can occur multiple times per month,” said Dr. John F. Tisdale, Chief of Cellular and Molecular Therapeutics Branch of the National Heart, Lung, and Blood Institute (NHLBI).
Read: For Sickle Cell Disease patients, every month is Sickle Cell Awareness Month
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