The oral medicine has been approved for the treatment of cystic fibrosis in young patients aged six to 11 years, who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Vertex Pharmaceuticals has secured approval from the European Commission for the label extension of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.
The oral medicine has been approved for the treatment of cystic fibrosis (CF) in young patients aged six to 11 years, who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The approval allows more than 1,500 children to gain eligibility for a treatment targeting the underlying cause of cystic fibrosis.
Cystic fibrosis is a rare, progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. The life-shortening genetic disease is estimated to affect more than 83,000 people globally.
The disease is caused by a defective and/or missing CFTR protein that results from certain mutations in the CFTR gene.
KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is said to increase the quantity and function of the CFTR protein at the cell surface.
Vertex CEO and president Reshma Kewalramani said: “We are delighted that KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now approved for these young patients in the European Union.
“It provides a new treatment option for physicians to help treat the underlying cause of this devastating disease early in life.
“This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF.”
By working together, elexacaftor and tezacaftor help to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein.
Known as a CFTR potentiator, Ivacaftor is designed to enable CFTR proteins to transport salt and water across the cell membrane.
According to the company, ivacaftor, tezacaftor and elexacaftor help in hydrating and clearing mucus from the airways, through their combined actions.
New Zealand and in Switzerland have also approved the medicine for people with CF aged six and above. It is known as TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in the two countries.