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New drug for cystic fibrosis approved by European regulators

Regulators in Ireland must now approve the drug ahead of negotiations between the drug’s manufacturer and the HSE regarding pricing.

Cystic fibrosis patient Malea Gardner with Child Life Specialist Catherine Coakley
Cystic fibrosis patient Malea Gardner with Child Life Specialist Catherine Coakley
Image: Fernando Salazar/AP/Press Association Images

A NEW DRUG that can help people with a certain form of cystic fibrosis has been approved by European regulators.

The European Medicines Agency approved the drug Kalydeco, manufactured by Vertex, which treats people with cystic fibrosis aged six and over who have at least one copy of the G551d mutation.

An estimated 1,100 people in Europe have the G551D mutation, with about 100 of those living in Ireland.

“The European approval of Kalydeco is an important step in our commitment to bring transformative new medicines to people with cystic fibrosis,” said Jeffrey Leiden, MD, PhD, Vertex’s Chair, President and Chief Executive Officer. “We’re preparing to supply pharmacies throughout Europe with Kalydeco and are working closely with national health authorities to make it available to patients as quickly as possible.”

Regulators in Ireland must now approve the drug ahead of any negotiations between Vertex and the HSE corporate pharmaceutical unit regarding pricing. The drug is now approved in both Europe and the United States.

“Cystic fibrosis is a life-threatening genetic disease that causes devastating effects, particularly in the lungs, including the build up of thick, sticky mucus which becomes infected and severely limits normal breathing,” said Stuart Elborn, MD, Kalydeco investigator and President of the European Cystic Fibrosis Society.

“Kalydeco is one of the most important advances in the treatment of cystic fibrosis since the discovery of the CF gene in 1989. By treating the underlying cause of the disease in people with the G551D mutation, Kalydeco helped them breathe more easily, gain weight and resulted in certain improvements in quality of life.”

Cystic fibrosis is Ireland’s most commonly inherited disease and Ireland has the highest proportion of people with cystic fibrosis in the world. Globally, 3-4 per cent of people with the condition carry the G551d gene mutation – but in Ireland the proportion is around 10 per cent, according to the Cystic Fibrosis Association of Ireland (CFAI). The proportion is greater in the south west of the country, with over 20 per cent of people carrying the G551d gene mutation in some places.

It is expected to take a number of weeks for Vertex to make a submission to the regulatory authorities in Ireland. The CFAI says it is looking for clarification from the National Centre for Pharmacoeconomics and the HSE regarding how long the approval process is likely to take, and says it will be seeking the process to be expedited as quickly as possible.

Read: Emergency services forced to abandon 65-mile kayak for Cystic Fibrosis>
Read: Why do females fare worse than males with cystic fibrosis?>
Read: 270,000 people in Ireland suffer rare health condition>

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