Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that
the European Committee for Medicinal Products for Human Use (CHMP) has
issued a positive opinion by consensus recommending the approval of
KALYDECO™ (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 treat the underlying cause of CF, 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 this mutation.
The CHMP opinion was based on positive findings from two global Phase 3
studies in which KALYDECO demonstrated unprecedented improvements in
breathing and other measures of disease for people ages 6 and older with
this specific genetic mutation. People treated with KALYDECO experienced
significant and sustained improvements in lung function, weight gain and
certain quality of life measurements compared to those on 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 adverse events
associated with KALYDECO were mild to moderate. Adverse events most
commonly observed in those taking KALYDECO included headache, upper
respiratory tract infection (common cold), stomach pain and diarrhea.