Vertex Pharmaceuticals has won approval in Europe for its next-generation cystic fibrosis therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), shoring up the company’s dominance in treating the rare and progressive genetic disease.
The approval by the European Commission (EC) follows a positive recommendation by the European Medicines Agency (EMA) in April.
Alyftrek will be available for patients aged six years and older with cystic fibrosis who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Alyftrek will provide a revenue safety net as Vertex braces for a loss of patent protection for blockbuster Trikafta (elexacaftor/tezacaftor/ivacaftor). Trikafta, known under the brand name Kaftrio in Europe, is the best-selling cystic fibrosis drug in the world, generating revenue of $11.02bn in 2024.
Kaftrio loses patient protection in 2037, at which point biosimilars are set to enter the market. Historically, this erodes the market dominance held by the branded drug. Alyftrek has patent protection until 2039.
Alyftrek demonstrated the same efficacy in cystic fibrosis treatment as measured by a lung parameter when pitted against Kaftrio in head-to-head trials. However, the new triple combination therapy was superior in reducing sweat chloride, demonstrating greater improvement in CFTR function, according to Vertex.
Along with an edge on certain measures of efficacy, Alyftrek has a dosing advantage. Unlike Kaftrio, which is taken twice daily, Vertex’s new product is taken only once per day.
Analysis by GlobalData’s Pharma Intelligence Centre forecasts Alyftrek will bring in sales of $6.1bn by 2031. Whilst models do not extend to 2037 – the year Kaftrio loses patent protection – data shows that full year revenue will already start falling from 2025, largely due to Alyftrek picking up market share. The pharma company has a market cap of $116bn.
Vertex’s CEO Reshma Kewalramani said: “Thousands of people with cystic fibrosis across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus Kaftrio.
“With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with cystic fibrosis.”
US market provides uptake hint
The US Food and Drug Administration (FDA) approved Alyftrek in December 2024, providing a window into market dynamics for the drug. Revenue for Alyftrek in its first full quarter of commercial availability in the US was $53.9m, below a William Blair estimate of $81.8m.
In a research note following Vertex’s Q1 results in May 2025, William Blair analyst Miles Minter said: “We continue to expect the switch from Trikafta to Alyftrek to be slower than the switch rates seen following historical CFTR modulator launches in Vertex’s portfolio, but we expect the company to see a successful switch over the long term and to benefit from midsingle-digit percentage margin improvements due to a favourable royalty structure with the Cystic Fibrosis Foundation.”
“Alyftrek also extends Vertex’s CFTR modulator composition-of-matter patent protection from 2037 for Trikafta [Kaftrio] into 2039 for Alyftrek,” Minter added.
