Seventy children with cystic fibrosis to benefit from new drug

The cystic fibrosis (CF) drug Orkambi is to be made available to about 70 children in Ireland following a deal between the manufacturer and the European Commission over its provision for two- to five-year-olds.

Availability of the drug, which is currently provided to patients aged six and older, is being extended after the Commission extended the label for Orkambi for the treatment of children with CF, aged two to five, who have two copies of the F508del mutation, the most common form of the disease.

Under a deal reached between manufacturer Vertex Pharmaceuticals and the HSE and Government in 2017, new treatments and extensions of existing treatments to new patients are made available to Irish CF patients. The drug was made available to six to 11-year-olds from March 2018.

Vertex says it will work with the HSE to securely timely access for the drug to the new cohort and says it expects this will happen shortly.

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The company claims that by treating the underlying cause of CF early, it may be able to modify the course of the disease and offer patients “the chance of improved outcomes”.

CF is an inherited disease that primarily affects the lungs and digestive system of about 1,300 children and adults in Ireland. It causes the body to produce a thick, sticky mucus that clogs the lungs and obstructs the pancreas. It also stops natural enzymes from helping the body break down and absorb food.

There is no cure. Ireland has one of the highest rate of CF in the world, with the incidence estimated at about four times the average for Europe.

A fight for access to the drug Orkambi ensued in 2017 after it was revealed Vertex was seeking €159,000 per patient for the drug, which the HSE estimated would cost some €400 million over five years.

Hundreds of people protested outside the Dáil, demanding access to the drug which was shown to improve patients’ lung function and reduce hospitalisation and exacerbations (a worsening of symptoms) by 40 per cent.

Respiratory physician at Beaumont Hospital Gerry McElvaney told The Irish Times last year the results from Orkambi were "better than expected" and had resulted in fewer hospitalisations for some patients. However, there was little improvement in terms of lung function and a small percentage of patients could not tolerate it at all.