Phase 3 study of ataluren for the treatment of nonsense mutation cystic fibrosis (nmCF)

PTC Therapeutics announced it has completed enrollment of a Phase 3 trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF). The 48-week study is designed to determine whether ataluren can improve lung function in patients with nmCF.  The trial has enrolled 238 patients at 36 sites in North America, Europe and Israel. The primary objective of the registration-directed, double-blind, placebo-controlled study is to determine whether ataluren can improve lung function in patients with nmCF, as measured by forced expiratory volume in 1 second (FEV1). Additional secondary endpoints are evaluating other aspects of patient function, drug activity, and safety.

Ataluren is a protein restoration therapy designed to enable the formation of full-length, functional cystic fibrosis transmembrane regulator (CFTR) protein in patients with cystic fibrosis due to a nonsense mutation.  CFTR is a critical protein lacking in CF patients.

For more information call (908) 222-7000 or visit www.ptcbio.com.