The US Food and Drug Administration (FDA) has approved voxelotor oral tablets (Oxbryta, Global Blood Therapeutics) for the treatment of sickle cell disease (SCD) in adults and children aged 12 years.

Voxelotor is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD.

The FDA approved voxelotor based on results of the phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization) clinical trial involving 274 patients with SCD patients randomly assigned to voxelotor or placebo.

Earlier this month, the FDA approved crizanlizumab (Adakveo, Novartis) to reduce the frequency of vaso-occlusive crisis in patients aged 16 years or older with sickle cell disease.

Leave a Reply

2020 © MedweiSer Health

error: Content is protected !!