Vertex wins European approval for new cystic fibrosis combination

European regulators have approved Vertex’s new cystic fibrosis (CF) combination treatment Symkevi.

The European Commission’s greenlight for Symkevi (tezacaftor/ivacaftor) will allow it to be used in combination with Kalydeco (ivacaftor) for patients aged 12-plus.

In doing so it will offer a new treatment option for individuals with two copies of the F508del mutation, the most common mutation in cystic fibrosis.

Symkevi has also been approved as a combination treatment for those who have one copy of the F508del mutation and a copy of one of 14 mutations in which the gene shows residual cystic fibrosis transmembrane conductance regulator (CFTR) activity.

The EU marketing authorisation was based on results from two phase 3 studies, which showed that treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function.

The combination had a generally well-tolerated safety profile and a lack of increased respiratory adverse events compared to a placebo.

Dr Reshma Kewalramani, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, said: “Today marks an important milestone for many CF patients in Europe, including those who so far have had no available option to treat the CFTR protein defect responsible for their disease.

“With [this] marketing authorisation (MA), we are rapidly moving towards treating 90% of CF patients.”

Tezacaftor/ivacaftor in combination with ivacaftor was approved by the US Food and Drug Administration (FDA) in February 2018 and by Health Canada in June 2018. It is marketed as Symdeko in North America.

Last month, Vertex met representatives of England’s cost watchdog NICE and representatives from NHS England to discuss access to its drug, Orkambi, which is the first medicine able to treat the underlying cause of CF in people aged six and over who have two copies of the F508del mutation.

A row over pricing for Orkambi began when NICE rejected it more than two years ago, leaving patients in limbo and sparking anger from the Cystic Fibrosis Trust, which is calling for Orkambi to be made available on the NHS as soon as possible.

Vertex wants the NHS to fund all its approved CF drugs – and any future medications in its pipeline – in a long-term deal, and claims that the health service is undervaluing its CF patients.

The UK has the second largest population of patients with CF in the world, with around 10,000 people affected by the debilitating condition.