Skip to main content

Many CF patients that take modulator regimens of the above dual combinations will need to transition to the triple combination. An assessment of whether adequate exposures to achieve clinical efficacy are maintained during this transition was needed, as this has not been directly addressed in clinical trials. PBPK modeling using Simcyp was used for this analysis, specifically to understand the CYP3A4 interactions during the CF transmembrane conductance regulator (CFTR) process. Individual models for each drug were developed, followed by simulations of various combination to assess exposure.

The PBPK modeling demonstrated that immediate transfer from the three dual combinations to the triple combination resulted in sustained CFTR in patients 12 years and higher. Clinical trials on younger patients are ongoing.

CS Body Content 1 Determining Exposures

“Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,”

- Acting FDA Commissioner Ned Sharpless, M.D. –FDA Press release** **

CF is an inherited condition that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems with digesting food. Symptoms usually start in early childhood and vary from child to child, but the condition gets slowly worse over time, with the lungs and digestive system becoming increasingly damaged.

CS Body Content 2 Determining Exposures

How Can We Help?