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CHMP Recommends Label Extension for Vertex Pharmaceuticals' Cystic Fibrosis Drug

NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use has recommended expanding the indication for Vertex Pharmaceuticals' Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor to include younger children with cystic fibrosis, a rare, progressive disease caused by certain mutations in the CFTR gene, the drugmaker said Friday.

The drug, an oral medication, was already approved in the EU for patients ages 6 years and older and who have at least one F508del mutation in the CFTR gene. Now, CHMP recommends expanding the indication for children ages 2 to 5 and with those same gene mutations.

Kaftrio is designed to increase quantity and function of the CFTR protein at the cell surface in patients with cystic fibrosis.   "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 2 years," Nia Tatsis, executive VP, chief regulatory officer, and chief quality officer at Vertex, said in a statement.

The US Food and Drug Administration approved Boston-based Vertex's drug, branded as Trikafta in the US, for patients as young as 2 in April.