Article
The U.S. Food and Drug Administration's Experience with Ivacaftor in Cystic Fibrosis. Establishing Efficacy Using In Vitro Data in Lieu of a Clinical Trial.
Annals of the American Thoracic Society - 1 Jan 2018
Durmowicz Anthony G, Lim Robert, Rogers Hobart, Rosebraugh Curtis J, Chowdhury Badrul A
Abstract excerpt
On May 17, 2017, the U.S. Food and Drug Administration expanded the patient population for use of ivacaftor to include patients with cystic fibrosis with relatively rare mutations in the cystic fibrosis transmembrane conductance regulator gene. The label expansion is unique in that clinical efficacy was not based on clinical data but on in vitro assay data demonstrating increased chloride ion transport across...
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