GBT announces early access program for voxelotor
US biopharma Global Blood Therapeutics (Nasdaq: GBT) has announced the initiation of an early access program for voxelotor in Europe and other regions outside the USA, for the treatment of hemolytic anemia in sickle cell disease (SCD) patients aged 12 years and older.
An oral, once-daily therapy, voxelotor directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anemia, which impairs adequate oxygen delivery to the tissues and organs in the body.
"The unmet need in this devastating disease is profound"Voxelotor is approved in the USA under the trade name Oxbryta for the treatment of SCD in patients aged 12 years and older. GBT previously announced it plans to seek regulatory approval to treat hemolytic anemia in SCD in patients in the same age range in Europe.
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