The Food and Drug Administration (FDA) has approved the first drug that target the underlying cause of the lung disease Cystic Fibrosis, a move that is being heralded by the Cystic Fibrosis Foundation.
The drug, developed by Vertex Pharmaceuticals Inc., and funded in large part by CF Foundation, will be sold under the brand name Kalydeco. It is indicated for a segment of CF patients ages 6 and older who have the G551D mutation of the disease. This mutation is present in roughly 4 percent of CF patients in the United States.
In people with this mutation, a defective protein caused by CF moves to its proper place at the surface of the cell but does not function correctly. Instead, the defective protein acts like a locked gate, preventing the proper flow of salt and fluid in and out of the cell. Kalydeco works by unlocking that gate and restoring the function of the defective protein.
The drug dramatically improves lung function, lowers sweat chloride levels and helps patients gain weight – all key clinical indicators of CF.
Nearly 90 percent of people with CF have another mutation, called Delta F508. Developing therapies for patients with the Delta F508 mutation is among the current research priorities of the CF Foundation. Progress toward this goal includes an ongoing Phase 2 clinical trial of Kalydeco in combination with another potential therapy, VX-809, in people with the Delta F508 mutation. Results from the first part of this Phase 2 trial were positive. The second part of the study is now under way.