Vertex Pharmaceuticals Incorporated has announced that the European Commission has approved KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages six 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 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. In people with the G551D mutation, KALYDECO helps the defective CFTR protein function more normally. An estimated 1,100 people in Europe have the G551D mutation. The approval of KALYDECO comes two months after the European Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion and is the first European approval for Vertex.
“The European approval of KALYDECO is an important step in our commitment to bring transformative new medicines to people with cystic fibrosis,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s Chair, President and Chief Executive Officer. “We’re preparing to supply pharmacies throughout Europe with KALYDECO and are working closely with national health authorities to make it available to patients as quickly as possible.”
The European Commission’s decision is based on positive findings from two global Phase 3 studies in which KALYDECO demonstrated significant and sustained improvements in breathing, weight gain and other measures of disease for people ages six and older with this specific genetic mutation, compared to placebo. In addition, people who took KALYDECO were 55% 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.
Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of the adverse events associated with KALYDECO were mild to moderate. Adverse reactions very commonly observed in those taking KALYDECO (>1/10) included headache; upper respiratory tract infection (common cold) including sore throat and nasal congestion; rash; diarrhoea; and abdominal pain (stomach ache). Two patients in the group receiving KALYDECO reported a serious adverse reaction of abdominal pain.
“Cystic fibrosis is a life-threatening genetic disease that causes devastating effects, particularly in the lungs, including the build up of thick, sticky mucus which becomes infected and severely limits normal breathing,” said Stuart Elborn, M.D., KALYDECO investigator and President of the European Cystic Fibrosis Society. “KALYDECO is one of the most important advances in the treatment of cystic fibrosis since the discovery of the CF gene in 1989. By treating the underlying cause of the disease in people with the G551D mutation, KALYDECO helped them breathe more easily, gain weight and resulted in certain improvements in quality of life.”
“KALYDECO is an exciting new beginning in the treatment of cystic fibrosis, but we’re not finished,” said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. “The scientists at Vertex, in collaboration with doctors, patients and advocates around the world, are working hard to develop additional new medicines to treat the underlying cause of the disease in many more people with cystic fibrosis.”
KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
KALYDECO™ (ivacaftor) is the first treatment to target the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. KALYDECO (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012, for use in people with CF ages six and older who have at least one copy of the G551D mutation in the CFTR gene.
Vertex retains worldwide rights to develop and commercialise KALYDECO. KALYDECO is under Priority Review by the Therapeutic Product Directorate (TPD) of Health Canada, and an application for review has been submitted to the Therapeutic Goods Administration (TGA) of Australia.