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Sickle hemoglobin is produced by a β-point mutation in the globin gene, which is less soluble than normal fetal or adult hemoglobin.
sickle cell disease (SCD) includes sickle cell anemia, sickle β thalt anemia, hemoglobin SC (HbSC) disease, etc.
The European Medicines Agency (EMA) has accepted a sales authorization application from the pharmaceutical company GBT for Oxbryta (voxelotor).
Oxbryta (voxelotor) is designed to treat hemolytic anemia in patients with sickle cell disease (SCD) aged 12 and over.
Oxbryta is a state-of-the-art oral therapy designed to inhibit hemoglobin polymerization, which can lead to sickle and destruction of red blood cells in SCDs, which in turn affect blood and oxygen flow throughout the body, while reducing the amount of oxygen delivered to tissues and organs, which can lead to life-threatening complications.
results from the HOPE Study no. III and the HOPE-KIDS 1 study in Phase II.
in the HOPE study, patients in the Oxbryta (voxelotor) group had significantly improved hemoglobin (Hb) levels at 24 weeks.
EMA has awarded Oxbryta Priority Medicines (PRIME). Ted Love, President and CEO of
GBT, said: "Sickle cell disease has a devastating impact on the lives of patients and their families, including serious and life-threatening complications that can lead to organ damage and premature death.
despite such a huge demand, there is currently no cure in Europe that can change the progress of the disease."
