Connections in Common
I read with interest the article by Doug Birch in the Winter 2017 issue concerning ivacaftor treatment for a cystic fibrosis patient with a rarer CF mutation [“A Genetically Unique CF Patient”]. It is wonderful that ivacaftor provided benefit to this patient who does not carry the G551D mutation.
Johns Hopkins has an additional connection with ivacaftor. I was chairman of the clinical pharmacology department at Vertex Pharmaceuticals and was a primary developer on the ivacaftor program. Together with my colleagues Juihong Zha and John Mondrick from the Metrum Research Institute, we modeled the dose response of ivacaftor and determined the dose and schedule. Dr. Zha and I wrote the clinical pharmacology sections of the first label and the documents for the regulatory filings.
Johns Hopkins was one of over 100 clinical trial sites where the drug was studied. The large number of sites was necessary because the particular CF mutation for which the drug is approved is not a common one among CF patients in a disease that is, itself, not common.