Vertex’s new cystic fibrosis treatment approved in EU

LONDON - Vertex Pharmaceuticals (NASDAQ:VRTX), a prominent biotechnology player with a market capitalization of $114.33 billion and strong financial health according to InvestingPro, announced Monday that the European Commission has approved ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor) for treating cystic fibrosis in patients aged 6 years and older with at least one non-class I mutation in the CFTR gene.

The approval makes ALYFTREK available to approximately 31,000 people with cystic fibrosis across the European Union, representing the broadest label for this medicine globally. This expansion aligns with Vertex’s impressive revenue growth of 9% over the last twelve months, reaching $11.1 billion.

In head-to-head clinical trials, ALYFTREK demonstrated non-inferiority to the company’s existing treatment KAFTRIO on lung function measurements while showing superior results in reducing sweat chloride levels, indicating greater improvement in CFTR protein function.

"Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care," said Professor Marcus A. Mall of Charité Universitätsmedizin Berlin, according to the press release statement.

Patients in Ireland, Denmark, and Germany will have access to the medication shortly after this approval due to existing reimbursement agreements and healthcare system provisions. Vertex stated it will continue working with reimbursement bodies across other EU member states to expand access.

Cystic fibrosis affects more than 109,000 people worldwide and is caused by defective or missing CFTR protein resulting from mutations in the CFTR gene. The disease affects multiple organs including the lungs, liver, pancreas, and digestive tract.

Vertex currently treats over 75,000 cystic fibrosis patients with its medicines in more than 60 countries, representing approximately two-thirds of diagnosed patients eligible for CFTR modulator therapy. With a healthy current ratio of 2.65 and strong market position, the company maintains robust operational flexibility. Discover more detailed financial metrics and 10+ additional ProTips with InvestingPro.

The medication is already licensed in the United States and United Kingdom, and remains under regulatory review in Canada, Switzerland, Australia, and New Zealand. Analysts maintain a positive outlook on Vertex’s growth trajectory, with price targets ranging from $330 to $621 per share, reflecting the market’s confidence in the company’s expansion strategy.

In other recent news, Vertex Pharmaceuticals has seen a series of developments that may interest investors. The company reported promising clinical data for its type 1 diabetes treatment, zimislecel, with William Blair reiterating an outperform rating due to successful insulin secretion and regulation in patients. H.C. Wainwright also maintained a Buy rating, highlighting that 83% of patients in a study achieved insulin independence. On the other hand, RBC Capital lowered its price target for Vertex to $420, citing slower uptake of the cystic fibrosis treatment Alyftrek, which could impact operating margins. BofA Securities reaffirmed a Buy rating with a $567 price target, noting positive competitor data that could favorably position Vertex’s povetacicept program. Meanwhile, BMO Capital maintained an Outperform rating and a $557 price target, focusing on the launch of Vertex’s new acute pain treatment, Journavx, and its prescription data. These updates reflect Vertex’s ongoing efforts to diversify its treatment portfolio and respond to market dynamics.

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