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Positive Opinion on Kaftrio to Treat Children with Cystic Fibrosis


European regulators have recently expressed a positive opinion regarding Vertex Pharmaceuticals’ groundbreaking drug, Kaftrio. This medication is specifically designed to address the needs of children suffering from cystic fibrosis.

Vertex Pharmaceuticals, based in Boston, announced on Friday that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has endorsed the extension of Kaftrio’s label. The new label will allow the drug to be used in combination with ivacaftor for the treatment of cystic fibrosis patients between the ages of two and five. These patients must possess at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

Nia Tatsis, Vertex’s Chief Regulatory and Quality Officer, expressed her satisfaction with this development: “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.”

This endorsement from European regulators marks a significant advancement in the treatment options available for young children with cystic fibrosis.

By Dean Seal

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