Vertex Secures FDA Approval for New Cystic Fibrosis Treatment Amid Slowing Trikafta Sales

Vertex Pharmaceuticals (NASDAQ: VRTX) has strengthened its grip on the cystic fibrosis (CF) market with the U.S. Food and Drug Administration’s approval of Alyftrek, a once-daily treatment for the rare genetic disorder. The decision, announced in December, also included an expanded label for Trikafta, the company’s top-selling CF therapy, now approved for children as young as two years old.

Cystic fibrosis is a hereditary disease caused by a defective protein that disrupts the flow of salt and water in and out of cells, leading to persistent lung infections and digestive complications. Trikafta, originally approved in 2019 for patients aged 12 and older, has been a cornerstone of Vertex’s CF portfolio, generating $10.24 billion in sales in 2024. However, with sales expected to decline slightly to $10.20 billion in 2025, and more sharply thereafter, the approval of Alyftrek provides a crucial new revenue stream.

Alyftrek offers a key advantage over Trikafta: a once-daily regimen instead of twice-daily dosing. It is also effective in 31 additional CF mutations. However, like Trikafta, Alyftrek carries a black box warning for potential liver toxicity. Analysts project Alyftrek will generate $974 million in its first year, with sales climbing to $7.3 billion by 2030.

While CF treatments accounted for 99% of Vertex’s revenue in 2024, the company is expanding beyond its core franchise. Its newly approved non-opioid painkiller, Journavx (suzetrigine), marks the first federal approval of its kind in more than two decades, as Vertex seeks to diversify its portfolio.