Vertex Pharmaceuticals Gets European Regulators' Nod for Cystic Fibrosis Drug

By Dean Seal

European regulators have adopted a positive opinion of Vertex Pharmaceuticals' drug Kaftrio, a treatment for children with cystic fibrosis.

The Boston-based drugmaker said Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use supports a label extension for Kaftrio to be used in combination with ivacaftor to treat cystic fibrosis patients between the ages of two and five who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

The combination regimen is already approved for the treatment of cystic fibrosis patients aged six and older.

"Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for Kaftrio to patients as young as two years," Chief Regulatory and Quality Officer Nia Tatsis said.

Write to Dean Seal at dean.seal@wsj.com

(END) Dow Jones Newswires

September 15, 2023 09:24 ET (13:24 GMT)

Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Nov 2023 to Dec 2023