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Vertex Announces Reimbursement Agreement in Spain for KAFTRIO in Combination With Ivacaftor

With this reimbursement agreement approximately 700 people with cystic fibrosis now have access to a CFTR modulator therapy for the first time.

Vertex Pharmaceuticals Incorporated today announced that the Spanish government has approved terms for the national reimbursement of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) for eligible patients. The agreement covers people with CF ages 12 years and older who have at least one copy of the F508del mutation, regardless of the other mutation type in the CFTR gene. KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor can be prescribed to eligible patients by treating physicians once the medicine is listed in the national Official Gazette Nomenclator.

“The formalized agreement is an important milestone for people living with cystic fibrosis in Spain. We are pleased that the Ministry of Health recognized the value of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor. Today’s announcement means that the medicine can be prescribed to eligible patients in Spain when listed,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We would like to thank the Ministry of Health for its collaborative approach, as well as the CF community for their important input during this process.”

With this agreement, Spain joins the group of over 25 countries — such as Ireland, Germany, Austria, Slovenia, Croatia, Luxembourg, Denmark, Finland, France, Portugal, Italy, Switzerland and the U.K. — where eligible patients with CF have access to the triple combination therapy.

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About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

About KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in A Combination Regimen With Ivacaftor
In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the CFTR gene.

Source: Vertex Pharmaceuticals
Date: Nov 19, 2021
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