The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.
This approval marks a significant milestone for Vertex Pharmaceuticals, making Alyftrek their fifth CFTR modulator to secure FDA approval, offering hope to an expanded group of CF patients.
CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. Without functional CFTR, thick mucus builds up in organs like the lungs, leading to chronic infections and lung damage.
The CFTR gene itself is complex, with over 2,000 known mutations. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation. As a result, the diversity of CFTR mutations presents significant challenges in developing therapies that can benefit all CF patients.
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Alyftrek targets the root cause of CF by enhancing CFTR protein function. It is the first CFTR modulator to offer once-daily dosing, simplifying treatment for patients who usually require multiple doses of other medications. This dosing schedule is a notable advantage over Vertex’s previous drug, Trikafta (elexacaftor/tezacaftor/ivacaftor), which requires multiple daily doses. While both drugs target the CFTR protein, Alyftrek expands treatment options by addressing 31 mutations unresponsive to Trikafta.
In clinical trials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels. Alyftrek also proved safe in younger patients aged six to 11 years. With the largest Phase III CF clinical trial program in history, involving over 1,000 participants across 20 countries, the results were promising, paving the way for broader use.
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Alyftrek will soon be available to US patients, offering a critical alternative to current therapies. Its once-daily dosing is a breakthrough for those managing complex regimens.
The diversity of CF treatments is expanding with investigational therapies such as Spirovant’s inhaled gene therapy SP-101 in a Phase I/II trial and Kither Biotech’s inhaled peptide therapy KIT2014 in a Phase I trial. While Alyftrek works by modulating CFTR to improve ion transport, SP-101 uses gene therapy to deliver a functional CFTR gene, potentially benefiting patients with mutations unresponsive to other CFTR modulators. KIT2014 targets both CFTR enhancement and inflammation, aiming to reduce the damage caused by inflammation in CF patients.
Vertex remains committed to advancing CF research, with plans to expand the impact of their CFTR modulators and explore new genetic mutations.
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