Vertex’s Orkambi can be used in even younger cystic fibrosis patients, says European Commission
Vertex has announced that the European Commission has decided to approve a label extension in the region for Orkambi (lumacaftor/ivacaftor) in the treatment of cystic fibrosis (CF), expanding the pool of eligible patients to include children aged between two and five years of age who have two copies of the F508del mutation.
The ruling follows a positive CHMP opinion for the therapy back in November. The drug was originally approved in Europe in the same indication for patients older than 12, with an extension to this coming in January 2018 to include paediatric patients aged between six and 11.
The latest decision was made on the back of Phase 3 data derived from 60 patients which demonstrated that Orkambi’s safety profile in these younger patients remained consistent with data observed in patients older than six, and that the therapy was generally well tolerated over a treatment duration of 24 weeks.
“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 Dr 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 technological pharmaceutical company IMIDEX has been granted clearance from the US Food and Drug …
On 16 August 2023, the US Food and Drug Administration (FDA) officially cleared Artiva Biotherapeutics’ …