Quick Facts
FONT-SIZE Plus   Neg
Share SHARE

EU Approves Vertex Pharma's KALYDECO For Cystic Fibrosis - Quick Facts

RELATED NEWS
Trade VRTX now with 

Vertex Pharmaceuticals Inc. (VRTX: Quote) said the European Commission has approved KALYDECOTM 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 or CFTR gene. KALYDECO is the first medicine to target the underlying cause of the disease in these patients.

Cystic fibrosis is a rare genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene.

KALYDECO helps the defective CFTR protein function more normally in patients with the G551D mutation. An estimated 1,100 people in Europe have the G551D mutation. The European Approval is based on positive findings from two global Phase 3 trials in which KALYDECO showed significant and sustained improvements in breathing, weight gain and other measures of disease for people ages 6 and older with this specific genetic mutation, compared to placebo.

Further, people who took KALYDECO were 55 percent less likely to have pulmonary exacerbations, or periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits, than those who received placebo, the company noted.

The KALYDECO approval comes two months after the European Committee for Medicinal Products for Human Use issued a positive opinion and is the first European approval for Vertex.

Register
To receive FREE breaking news email alerts for Vertex Pharmaceuticals Inc. and others in your portfolio

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Policymakers of Reserve Bank of Australia decided to maintain its record low interest rate once again as they continue to assess that the most prudent course is a period of stability in interest rates. The monetary policy board retained the cash rate at 2.50 percent. The rate has been at the current level since August 2013. A ban on athletes using stamina-boosting gases - xenon and argon - came into effect on Monday. The World Anti-Doping Agency (WADA) said Hypoxia-Inducible Factor (HIF) activators Xenon and Argon have been added to the 2014 List of Prohibited Substances and Methods List following the required three-month notice period and UNESCO's communication to all States Parties. A recommendation to revise the British manufacturing growth eased further in August to its lowest level in 14 months as output and demand increased at slower rates, survey results from Markit Economics showed Monday. The Markit/CIPS Purchasing Managers' Index dropped to 52.5 from a revised 54.8 in July. Economists had expected the score to fall to 55.1 from July's original figure of 55.4.
comments powered by Disqus
FREE Newsletters, Analysis & Alerts

 

Stay informed with our FREE daily Newsletters and real-time breaking News Alerts. Sign up to receive the latest information on business news, health, technology, biotech, market analysis, currency trading and more.