Vertex secures EU approval for Alyftrek, strengthening cystic fibrosis position

Vertex Pharmaceuticals has gained approval from the European Commission for its innovative cystic fibrosis drug, Alyftrek, which is expected to strengthen the company's position in treating this serious genetic disorder. Alyftrek, a combination of deutivacaftor, tezacaftor, and vanzacaftor, will be available to individuals aged six and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The approval follows a positive recommendation by the European Medicines Agency in April, marking a significant step for Vertex as it prepares for the eventual loss of patent protection for its leading cystic fibrosis therapy, Trikafta (also known as Kaftrio in Europe). Trikafta, currently the world’s top-selling cystic fibrosis drug, is expected to generate $11.02 billion in revenue in 2024. However, its patent protection will expire in 2037, allowing biosimilars to enter the market and potentially erode its dominance. In contrast, Alyftrek is protected until 2039, providing a strategic advantage.

Alyftrek has shown comparable effectiveness to Kaftrio in treating cystic fibrosis, as determined by lung function parameters. Additionally, the newer medication demonstrated superior results in reducing sweat chloride, which indicates improved CFTR function. This efficacy advantage, coupled with a more convenient dosing regimen of once daily compared to Kaftrio's twice-daily requirement, positions Alyftrek favorably in the market.

According to GlobalData’s Pharma Intelligence Centre, Alyftrek is projected to achieve $6.1 billion in sales by 2031. Although forecasts do not extend to 2037, the year Trikafta loses patent protection, data indicates that full-year revenue for Trikafta will begin to decline starting in 2025 as Alyftrek captures a larger share of the market. With a market capitalization of $116 billion, Vertex is well-positioned to capitalize on this shift.

Vertex CEO Reshma Kewalramani expressed optimism, stating, “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.”

In the US, Alyftrek received approval from the Food and Drug Administration in December 2024. Initial revenue figures for Alyftrek in its first full quarter of commercial availability reached $53.9 million, which was below the projected $81.8 million by financial services firm William Blair. Analyst Miles Minter noted that while the transition from Trikafta to Alyftrek is expected to be slower than previous CFTR modulator switches in Vertex’s portfolio, the long-term outlook is favorable, with expected benefits from improved margins due to a beneficial royalty structure with the Cystic Fibrosis Foundation. Minter also highlighted that Alyftrek extends Vertex’s CFTR modulator patent protection from 2037 for Trikafta to 2039 for Alyftrek.

Overall, Vertex’s strategic moves with Alyftrek aim to maintain its leadership in cystic fibrosis treatment, ensuring continued innovation and market presence in the face of impending patent expirations for its blockbuster drug, Trikafta.