FDA approves new cystic fibrosis therapy
Av Cystic fibrosis is a hereditary and lifelong disease that leads to mucus formation in the throat, difficulty breathing, reduced lung function, and often premature death. The disease is linked to mutations in a gene that codes for a chloride channel and has been named cystic fibrosis transmembrane regulator (CFTR). In Sweden, approximately 700 people suffer from the disease.
The now FDA-approved combination treatment is called Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) and has been developed by the American company Vertex Pharmaceuticals. The market approval applies to the treatment of patients aged six years and older with at least one F508del mutation or another mutation in the CFTR gene that responds to Alyftrek – including 31 mutations that do not respond to previous CFTR modulators.
Vertex has previously developed several drugs targeting cystic fibrosis. The most well-known is Kaftrio, which is marketed in the U.S. under the name Trikafta. After a lengthy process, this treatment is now included in the high-cost protection for Swedish patients with cystic fibrosis.
Unlike Kaftrio, which is taken twice a day, Alyftrek is a once-daily drug.
“Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function — Alyftrek, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than Trikafta, is another step in achieving this goal”, says Vertex CEO Reshma Kewalramani in a statement.
Alyftrek is also being evaluated by regulatory authorities in the EU and several other markets.
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