This required us to develop a model using the in vitro and clinical data in healthy volunteers, verify with independent clinical data sets, create a new population file for SCD, and verify with adults and adolescents with the disease in order to predict exposure in children.

For predicting DDI with CYP3A4 enzymes, there were no clinical DDI studies conducted for us to use in building the model. To address this issue, we leveraged the model we built for dose prediction in healthy and SCD patients along with in vitro data to create the DDI predictions. We then performed a sensitivity analyses under multiple scenarios and were able to inform the final drug label without the need for any clinical studies. Further, there was no post-marketing requirement covering DDI.

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.

- FDA Press Release** **Ref: US FDA press release, “FDA approves novel treatment to target abnormality in sickle cell disease,” 2019 年 11 月 19 日

SCD is a group of inherited red blood cell disorders. The most common genetic disease in the world, approximately 250 million people worldwide carry the gene responsible for sickle cell disease and other hemoglobin diseases. Until recently, the only cure for SCD was a bone marrow or stem cell transplant.

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