CONTEXT: New data being presented at the European Hematology Association (EHA) 2021 Virtual Congress on the treatment of sickle cell disease with Oxbryta (voxelotor). Two real-world evidence studies support the results from the phase 3 study that treatment in younger patients are seeing much better outcomes in this life-long, debilitating disease. The real-world studies comprised one retrospective chart review and one prospective registry. GBT are continuing the registries in an effort to gain a deeper understanding of the long-term safety and efficacy of their compound. | US only data
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1. “New Data Analysis from Phase 2a HOPE-KIDS 1 Study (Oral Abstract #S260). A new analysis of data from 45 children with SCD ages 4 to 11 years enrolled in the open-label Phase 2a HOPE-KIDS 1 Study (GBT440-007) showed that treatment with Oxbryta (1,500 mg or weight-based equivalent dispersed in a pediatric-appropriate formulation) resulted in rapid and sustained improvements in hemoglobin.”
2. “Two Real-world Data Studies Demonstrated Sustained Improvements in Hemoglobin with Oxbryta Consistent with Results from the Phase 3 HOPE Study. SOUTH SAN FRANCISCO, Calif., June 11, 2021 (GLOBE NEWSWIRE) — Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced new data from the Phase 2a HOPE-KIDS 1 Study that showed children with sickle cell disease (SCD) ages 4 to 11 years treated with Oxbryta® (voxelotor) tablets experienced significant improvements in hemoglobin levels.”
3. ““The encouraging results seen with Oxbryta treatment in younger patients living with sickle cell disease support our belief that Oxbryta can have a positive impact across a wide age range of people living with this devastating, lifelong disease by reducing the sickling and destruction of red blood cells, thereby improving anemia and hemolysis – hallmarks of the condition,” said Ted W. Love, M.D., president and chief executive officer of GBT.”
4. “The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.4-7. About Oxbryta® (voxelotor) tablets. Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD).”
5. “The company has introduced Oxbryta® (voxelotor) tablets, the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD.”