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Vertex Announces EMA Type II Variation Marketing Authorization Application Validation For KAFTRIO

Vertex Pharmaceuticals Inc. (VRTX) announced Monday that the European Medicines Agency or EMA has validated a Type II Variation Marketing Authorization Application or MAA for the expanded indication of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor.

It is to treat Cystic Fibrosis or CF in patients ages 12 years and older with at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene. CF is a rare, life-shortening genetic disease.

If approved, eligible patients who have one copy of the F508del mutation and another CFTR mutation, such as a gating (F/G) or residual function (F/RF) mutation, will also be eligible for treatment.

The company noted that the MAA is supported by positive results from the global Phase 3 study with KAFTRIO announced in July 2020.

The Committee for Medicinal Products for Human Use or CHMP will now review the application and will issue an opinion to the European Commission regarding the potential approval for these patients.

KAFTRIO in combination with ivacaftor is currently approved in Europe to treat people with CF ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.

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