Make that 2 new sickle cell disease blockbuster hopefuls OK’d by the FDA in just a few days
Just days after Novartis scored a ‘breakthrough’ OK with their new drug crizanlizumab (Adakveo) to prevent vaso-occlusive crises (VOCs) among sickle cell disease patients, Global Blood Therapeutics $GBT has followed up and nabbed their own regulatory green light in the field.
Based on data from a study demonstrating a 51% increase in the rate of hemoglobin response among patients — compared to only 6.5% in the placebo group — regulators stamped an approval on Oxbryta, better known until now as voxelotor. The company calls this drug “transformative.” But the therapy failed to show a statistically significant improvement in the rate of VOCs, which is what sends patients to the hospital.
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