Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies
In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Voxelotor is an oral therapy taken once daily, it is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. Voxelotor was developed under FDA’s accelerated review and orphan designations. As drug types become more complex, we are using physiologically-based pharmacokinetic (PBPK) modeling and simulation to answer difficult development questions, such as in the case of voxelotor. Delivered via multiple pathways, our initial goal was to determine dose projections for children aged 9 months to 12 years which was followed by a request to predict drug-drug interactions (DDI) with CYP3A4 enzymes.
“Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder.”