FONT-SIZE Plus   Neg

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

Vertex Pharmaceuticals Inc. (VRTX) 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.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Apple confirmed that it has bought face animation software firm Faceshift, although it was tight lipped about how it intends to use the new addition. The move is speculated as a bid to expand into virtual reality. Environment ministry of South Korea Thursday ordered Volkswagen Korea to recall 125,522 diesel vehicles on determining actual emissions of nitrogen oxides. The ministry has asked the German automobile maker to pay a fine of 14.1 billion Won or $12.31 million. The company has to submit a recall plan by January 6, 2016. The U.S. Food and Drug Administration has alerted health care professionals and patients of a voluntary recall of compounded multivitamin capsules containing high amounts of Vitamin D3 (Cholecalciferol), distributed nationwide by Glades Drugs in Pahokee, Florida. FDA has received reports of several...
comments powered by Disqus
Trade VRTX now with 
Follow RTT