Vertex Kalydeco Gets Australian Regulatory Approval

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) Tuesday said the Therapeutic Goods Administration of Australia has approved Kalydeco (ivacaftor) for people with cystic fibrosis ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator(CFTR) gene.

Kalydeco is the first medicine to treat the underlying cause of the disease in these patients. Cystic fibrosis is a rare genetic disease caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Kalydeco is the first Vertex medicine approved in Australia.

"The approval of Kalydeco in Australia is an important milestone for the cystic fibrosis community," said Simon Bedson, Senior Vice President of International Commercial Operations for Vertex. "The rapid review and approval by the TGA is a major step in making Kalydeco available for eligible Australians."

The approval of Kalydeco was based on data from two global Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with Kalydeco experienced significant and sustained improvements in lung function and weight gain compared to those who received placebo.