Vertex’s CF drug gets label extension as pricing row continues

While UK cystic fibrosis patients continue their long wait for access to Vertex’s Orkambi, the drug has received a label extension from the European Commission allowing it to be used in younger children. The Commission has approved the drug for the treatment of children with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease. Previously, this indication was only approved for patients aged 6 and above.
“Today’s approval by the European Commission brings us one step closer to our goal of bringing treatment to all people living with CF,” said Reshma Kewalramani, executive vice president, global medicines development and medical affairs and chief medical officer at Vertex. ‘‘By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.’’ The label update is based on data from a phase 3 open-label safety study in 60 patients that showed treatment with Orkambi was generally well tolerated for 24 weeks, with a safety profile in these pediatric patients generally consistent with that in patients aged 6 years and older.