FDA expands Oxbryta approval for sickle cell disease
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The FDA granted accelerated approval to voxelotor tablets for the treatment children aged 4 to 11 years with sickle cell disease.
The agency previously approved the therapy to treat patients aged 12 years or older.
Voxelotor (Oxbryta, Global Blood Therapeutics) — an oral agent taken once daily — was the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of sickle cell disease.
The therapy restores patients’ red blood cells to a normal shape, reducing damage to red cells and the body’s tissues.
The FDA based the new indication on results of a phase 2 trial that included 45 children aged 4 to 11 years with sickle cell disease.
Study participants received voxelotor tablets for oral suspension based on body weight at the start of the trial.
Increase of hemoglobin greater than 1gram per deciliter from the start of the trial to week24 served as the main efficacy measure.
More than one-third (36%) achieved this outcome.
The accelerated approval requires that an ongoing study of voxelotor be completed to confirm that the hemoglobin increase results in clinical benefit, according to an FDA-issued press release.
The most common adverse events associated with voxelotor treatment include headache, diarrhea, abdominal pain, nausea, rash and fever.
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