New Drug for Cystic Fibrosis Receives Priority Review

As reported in Cystic Fibrosis News Today, a new drug combination created for Cystic Fibrosis, a mix of Vertex’s elexacaftor, tezacaftor, and Kalydeco has been granted priority review. The new mix of medications is intended for those who are not responding to the medications already available, rather than providing a new option for everyone with the disease. Both trials showed improvements in the patients lung functioning, less pulmonary exacerbations, and less chloride.

What is Cystic Fibrosis

Cystic Fibrosis is a disease that affects the digestive and respiratory systems. It causes people to produce too much mucus that causes blockages, trapping bacteria and preventing the healthy flow of air, nutrients, and typical movement through their systems. This makes those with the genetic condition suffer from breathing related difficulties and get more infections from the build up of bacteria.

The Drug’s Results

The drug has just been through two sets of phase three trials, each with promising results. The two sets of trials, AURORA F/MF and F/F, we’re done on two different mutations; F/MF on 403 people with one F508del mutation and one minimal function mutation, and F/F trials on 107 people with two F508del mutations. The drug combination is highly anticipated because it is the first drug that can be used for a particular type of cystic fibrosis, for those with a F508del mutation and one minimal function mutation.

The drug will receive its final clearance  on March 19, 2020 by the FDA.




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