CF drug approved for young children

Previously only available to those over 12
  • Deborah Condon

Cystic Fibrosis Ireland and the Minister for Health, Simon Harris, have welcomed an announcement that use of the drug, Orkambi, is to be extended to children with the condition who are aged between six and 11 years.

Vertex Pharmaceuticals, which manufactures the drug, has received EU approval, which will allow it to be used in younger children. While the drug is currently reimbursed here, until now, it was only licensed for use in children aged 12 and older.

Cystic fibrosis (CF) is Ireland's most common life-threatening inherited disease. There are around 1,200 people in Ireland with the condition and this country has the highest prevalence and the most severe types of the disease in the world.

The condition affects the glands, damaging many organs including the lungs, pancreas, digestive tract and reproductive system, impacting breathing, digestion and reproduction. It causes thick sticky mucus to be produced, blocking the bronchial tubes and preventing the body's natural enzymes from digesting food.

Many people with CF experience exacerbations of the condition, however Orkambi has been shown to reduce exacerbations and hospitalisations by up to 40% in those eligible to take it.

Almost half of people with CF are eligible to take this drug, which is aimed at those with a specific genetic mutation.

In a statement, Vertex said that the European Commission ‘has granted extension of the marketing authorisation for Orkambi'.

"The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to Orkambi," commented Simon Bedson, international general manager at Vertex.

The company added that it will ‘work closely with the HSE and relevant officials to secure timely access' for affected patients, and it hopes the drug will be made available ‘shortly'.

Minister Harris described the announcement as ‘good news for CF patients'. He said that the HSE will be putting in place the necessary reimbursement arrangements over the coming weeks for children aged between six and 11.

Cystic Fibrosis Ireland said that it understands that the HSE is aiming to make Orkambi available to eligible children ‘from the beginning of February 2018'.

"This is very welcome and we thank the HSE and the Minister for Health for their ongoing commitment to CF care in Ireland," added the organisation's CEO, Philip Watt.

For more information on Cystic Fibrosis Ireland, click here


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