New drug for cystic fibrosis


A DRUG that could benefit up to 100 people in Ireland with a certain type of cystic fibrosis has been approved by the European Medicines Agency.

The agency approved Kalydeco to treat people with the condition who have at least one copy of the G551d mutation, otherwise known as the Celtic gene. Cystic fibrosis (CF) is the most common genetically inherited disease and Ireland has the highest proportion of cystic fibrosis in the world.

The drug has to be approved here by regulators before negotiations can take place between the manufacturers Vertex and the HSE corporate pharmaceutical unit on the price of the drug.

Philip Watt, the chief executive of the Cystic Fibrosis Association of Ireland, urged the HSE to facilitate this approval process.

“It’s an exciting development,” he said. “The tablet can be taken orally, which is great because some CF treatments can take hours in hospitals. The people who took the drug in trials have reported an increase in lung function.”

In Ireland 10 per cent of the CF population could benefit from this drug as opposed to 4 per cent of the CF population in the rest of the world.