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Vertex Pharmaceuticals Cystic Fibrosis Drug Gets Positive Opinion in EU For Treatment in Infants

By Adriano Marchese


Vertex Pharmaceuticals said Friday that its treatment for cystic fibrosis was given a positive opinion by European Union regulators to treat infants with specific mutations of the gene.

The pharmaceutical company said that the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion for the label expansion of the drug ivacaftor, also known as Kalydeco.

The approval is to expand the treatment of infants with cystic fibrosis between the ages of one to four months who have certain specific mutations of the cystic fibrosis transmembrane conductance regulator gene.

Kalydeco aims to treat the underlying cause of cystic fibrosis, a life-shortening genetic disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.

In the EU, the treatment is already approved to treat the disorder in patients aged four months and older.


Write to Adriano Marchese at


(END) Dow Jones Newswires

February 23, 2024 07:59 ET (12:59 GMT)

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