Orkambi approval in Europe will extend treatment to more CF patients

Vertex's second drug for cystic fibrosis (CF), Orkambi, has been approved in Europe, opening up treatment to thousands more patients.

The new approval follows the 2013 launch of Kalydeco (ivacaftor), which was the first drug to act directly on the causes of the disease. Orkambi combines ivacaftor with another agent, lumacaftor, and could help around 12,000 people with CF aged 12 and older who have two copies of a particular gene mutation.

The EU approval makes Orkambi the first ever drug to treat the underlying cause of CF in patients who have two copies of the F508del mutation.

The approval is based on data from two phase III studies, TRAFFIC and TRANSPORT, and additional interim 24-week data from the subsequent extension study, PROGRESS, in people ages 12 and older who have two copies of the F508del mutation and were already being treated with standard-of-care medicines.

The studies showed patients taking Orkambi experienced significant improvements in lung function, as well as improvements in body mass index (BMI). People with CF frequently suffer from dangerous pulmonary exacerbations (acute lung infections), which can require hospitalisation and intravenous antibiotic use and these, too, were reduced.

High cost

Vertex says it will now begin the country-by-country reimbursement process, but hasn't given a clue to its price in these markets.

Vertex has priced the drug at around $259,000 per patient annually in the US, which has attracted controversy – despite this being lower than the price of Kalydeco. This lower price is because the drug can treat a significantly larger sub-group of CF sufferers.

The European price is likely to be significantly lower, but may still attract strong resistance from payers in Europe, where most CF patients do not have access to Kalydeco.

Jeffrey Leiden, Vertex's chairman, president and chief executive said the European approval was a major milestone in the firm's efforts to tackle the cause of CF.

"This has been 17 years in the making and is the result of a collaborative effort with the CF community. Our goal now is to help ensure that those who need this medicine can get it, and to continue our work for the two out of three people with cystic fibrosis in Europe who still don't have a treatment that addresses the underlying cause of their disease."