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EMA recommends breakthrough genetic treatment for cystic fibrosis

Vertex Pharmaceuticals’ Kalydeco would be first drug to treat cause of condition if approved

An advisory committe to the European Medicines Agency (EMA) has recommended Vertex Pharmaceuticals' Kalydeco for the treatment of cystic fibrosis in patients age 6 years and older who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene.

If approved by the European Commission (EC), it would be the first drug available in Europe to target the cause of cystic fibrosis rather than its symptoms.

Kalydeco (ivacaftor), which is taken twice-daily in tablet form, is a personalised medicine that treats the genetic disorder by restoring the function of the mutated CFTR protein.

This gene regulates salt and water transport in the body, but if it malfunctions, too much salt and water can be held in cells, leading to a build up of a thick mucus in the body's various passageways.

Current medications to treat cystic fibrosis include bronchodilator drugs to help open airways and DNase to break down mucus, but Kalydeco is the first to target the actual mechanism behind the condition.

The recommendation from the EMA's CHMP is based on two global phase III studies that demonstrated Kalydeco led to improvements in breathing and weight gain in people aged 6 years and older with the G551D genetic mutation.

It is estimated that about 1,100 people in Europe with cystic fibrosis have this specific mutation. The EMA has therefore designated the drug an orphan medication due to the limited number of people it could be used to treat.

Kalydeco was also reviewed through the EMA's accelerated assessment, which aims to speed up access to new medicines that are of major public health interest

Even with this accelerated process, the recommendation comes almost four months after Kalydeco was cleared by the US Food and Drug Administration (FDA), with the US agency taking just three months to review Vertex' marketing application.

This backs outcomes from a recent report that described how the FDA had a faster approval process than the EMA.

28th May 2012

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