Vertex Pharma: EU Oks KAFTRIO In Combination With Ivacaftor To Treat Children With Cystic Fibrosis

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Vertex Pharmaceuticals Inc. (VRTX) announced Tuesday that the European Commission has granted approval for the label extension of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.

The approval is for the treatment of cystic fibrosis or CF in patients ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene.

CF is a rare, life-shortening genetic disease, caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. It is a progressive disease, in which symptoms and organ damage manifest very early in life.

Reshma Kewalramani, Chief Executive Officer and President at Vertex, said, “This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF.”

The company noted that as a result of long-term reimbursement agreements in Austria, Northern Ireland and Denmark, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor shortly following regulatory approval by the European
Commission.

Vertex said it will continue to work with reimbursement bodies across the European Union to ensure access for all eligible patients.

The medicine has also been approved by regulatory authorities in New Zealand and in Switzerland, where it is known as TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), for people with CF ages 6 and above.

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