The FDA/ASH workshop is finished. Investors responded quite positive to it with prices spiking on significant volume:
If you didn't get a chance to listen in, I was, fortunately, able to hear every word. Below, I present the three key takeaways I had from the workshop.
Takeaway 1: "Hemoglobin" Is An Established Biomarker
During the second day of the workshop, "hemoglobin" was mentioned at nauseam. In particular, hemoglobin was listed as key biomarkers for hematological and neurological endpoints:
What is a "biomarker", as defined by the FDA?
a biomarker is a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes, or biological responses to a therapeutic intervention. An example that is familiar to many people is the use of blood glucose levels to measure the effectiveness of a diabetes medication. In this case, glucose is the biomarker.
Recall the requirements of accelerated approval:
The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit.
Global Blood Therapeutic's (GBT) voxelotor has demonstrated a robust effect on hemoglobin. Additionally, they have gathered TCD velocity data on patients before and after the use of voxelotor. As a result, GBT believes hemoglobin is a valid surrogate endpoint reasonably likely to predict clinical benefit:
Takeaways 2 & 3
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