CHMP positive opinion for Orkambi (lumacaftor/ivacaftor) in children with cystic fibrosis ages 1 to <2 years old
Vertex Pharmaceuticals announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of Orkambi(lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to less than 2 years old who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common form of the disease
“Symptoms and organ damage start very early in the lives of people with cystic fibrosis. Treating patients as young as possible is important, as it can potentially slow the progression of this devastating disease,” said Fosca De Iorio, Vice President, International Medical Affairs at Vertex. “Today’s news provides great hope that, if approved, a treatment option will be available for the first time for about 300 young children with CF who have two copies of the F508del mutation.”
In the EU ,Orkambi(lumacaftor/ivacaftor) is already approved for the treatment of people with CF who have two copies of the F508del mutation, ages 2 years and above.
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