Now Approved: A New Way to Treat Sickle Cell Disease in Children Ages 4 to Less Than 12
December 20, 2021
The US Food and Drug Administration (FDA) has approved Oxbryta for the treatment of sickle cell disease in patients ages 4 to less than 12 who weigh between 22 and 88 lbs (10 to 40 kg), according to GBT (Global Blood Therapeutics).
What is OXBRYTA?
OXBRYTA is a prescription medicine used for the treatment of sickle cell disease in adults and children 4 years of age and older.
It is not known if OXBRYTA is safe and effective in children with sickle cell disease below 4 years of age.
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
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"For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder. 'It is just amazing how far things have come,' says Victoria Gray, 34, of Forest, Miss. 'It is wonderful,' she told NPR in an exclusive interview after undergoing the landmark treatment for sickle cell disease. Gray is the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease."
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"The FDA granted priority review to crizanlizumab as a treatment for preventing painful, life-threatening events in sickle cell disease patients."
Learn more about crizanlizumab here: https://sicklecellanemianews.com/crizanlizumab-seg101/
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"In the study, voxelotor provided a rapid, statistically significant and sustained improvement in hemoglobin levels and reduced the incidence of worsening anemia and hemolysis."
Global Blood Therapeutics: New Drug Voxelotor In Clinical Trials
We are passionate and committed to developing new therapies that can provide transformative clinical benefit to patients.
Our late-stage product candidate voxelotor (GBT440) is an oral, once-daily therapy that is designed to modulate hemoglobin affinity for oxygen and is in clinical development for the treatment of sickle cell disease.