EC approves Orkambi for infants with cystic fibrosis

The European Commission has granted approval for the label extension of Orkambi (lumacaftor/ivacaftor), from US biopharma firm Vertex Pharmaceuticals (Nasdaq: VRTX), for the treatment of children with cystic fibrosis (CF) ages one to F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common form of the disease.

“This approval will offer some of the youngest children with cystic fibrosis the chance of improved outcomes, by treating their disease at a young age,” said Dr Carmen Bozic, executive vice president, lobal medicines development and medical affairs, and chief medical officer, Vertex. “With this important milestone, we move ever closer to our goal of providing medicines that treat the underlying cause of CF to all people living with the disease,” she added.

“CF symptoms and organ damage can manifest very early in life, so it is crucial to start treatment as early as possible,” said Dr Silvia Gartner, specialist in pediatrics and pneumonology, coordinator of the Pediatric Cystic Fibrosis Center, Barcelona. “Today’s approval provides us with a medicine that gives a window of opportunity to possibly delay the onset of CF for these very young eligible children,” she noted.