The European Commission approved the Vertex Pharmaceuticals drug 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 helps the defective CFTR protein function more normally in people with the G551D mutation, including an estimated 1,100 people in Europe. According to Vertex, Kalydeco is the first medicine to target the underlying cause of the disease in these patients, defective or missing CFTR proteins stemming from mutations in the CFTR gene.
The EC approval is Vertex’s first, and came two months after the European Committee for Medicinal Products for Human Use issued a positive opinion. The EC based its decision on positive findings from two global Phase III 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. Also, people who took Kalydeco were 55% less likely than those who received placebo to have pulmonary exacerbations, or periods of worsening in signs and symptoms of the disease that often require treatment with antibiotics and hospital visits.
Fewer people in the Kalydeco treatment groups discontinued treatment due to adverse events than in the placebo groups. Adverse reactions very commonly observed in those taking Kalydeco 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.
Kalydeco was discovered under a collaboration with Cystic Fibrosis Foundation Therapeutics, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
FDA approved Kalydeco in January. The drug is under Priority Review in Canada, while an application for review has been submitted in Australia.