Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has approved KALYDECO TM (ivacaftor) for people with cystic fibrosis (CF) 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 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 6 and older with this specific genetic mutation, compared to placebo. In addition, 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.
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.