Skip to content

Vertex gets FDA OK to expand users of Kalydeco in cystic fibrosis

August 1, 2017

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved KALYDECO® (ivacaftor) for use in more than 600 people with cystic fibrosis (CF) ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This approval was based on Phase 3 clinical data for KALYDECO in these mutations and follows the FDA’s approval of KALYDECO in May 2017 for 23 other residual function mutations, which was based on analyses of in vitro data. Both approvals are supported by more than five years of real-world clinical experience that demonstrate KALYDECO’s established safety and efficacy profile. Based on today’s approval, Vertex increased its guidance for 2017 KALYDECO product revenues to a range of $770 million to $800 million. Vertex’s guidance range for total CF product revenues in 2017 is now $1.87 billion to $2.1 billion, including ORKAMBI guidance of $1.1 billion – $1.3 billion.

“In the five years since KALYDECO became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease.”

CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt (sodium and chloride) and water into or out of cells in a number of organs, including the lungs. The five mutations covered under today’s approval (2789+5G—>A, 327226A—>G, 3849+10kbC—>T711+3A—>G, and E831X) cause CF and result in a moderate loss of chloride transport. People who have these mutations generally experience progressive lung function decline and other complications of the disease. All five of these mutations were evaluated as part of the previously disclosed Phase 3 EXPAND study in which the KALYDECO monotherapy arm met its primary efficacy endpoint and was generally well tolerated.

KALYDECO is now approved in the U.S. to treat people with CF ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.

http://bit.ly/2hjZLSr

Advertisements

From → Uncategorized

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: