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VRTX Secures Nod for Expanded Use of CF Drug Kaftrio in Europe

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Vertex Pharmaceuticals (VRTX - Free Report) announced that the European Commission (EC) has expanded the label of its blockbuster cystic fibrosis (CF) drug, Kaftrio, to treat all patients aged two years and older who have at least one non-class I mutation in the CFTR gene.

This approval was expected as the EMA’s Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending Kaftrio’s approval in February for the above indication. With this expanded label, nearly 4,000 more CF patients living in the region are now eligible for treatment with the drug.

Kaftrio is already approved for use in CF patients aged two years and older who have at least one F508del mutation in the CFTR gene.

The drug is marketed by Vertex for similar indications in the United States under the trade name Trikafta.

VRTX Stock’s Performance

Year to date, shares of Vertex have gained 18% against the industry’s 7% decline.

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Vertex’s Strong CF Portfolio

Vertex holds a dominant position in the CF market. It was the first company to successfully develop a drug that treats the underlying cause of CF. In addition to Trikafta/Kaftrio, the company markets four other CF medications — the recently approved Alyftrek, Symdeko/Symkevi, Orkambi and Kalydeco.

Vertex’s CF sales continue to grow, driven by Trikafta/Kaftrio, which added $10.24 billion in product sales in 2024. Sales of the drug were up about 15% year over year, driven by its continued robust performance across all markets.

The company aims to have small molecule treatments for most people with CF. In this regard, Vertex is developing an mRNA therapeutic, VX-522, in partnership with Moderna (MRNA - Free Report) for approximately 5,000 people with CF who do not make CFTR protein and cannot benefit from its CFTR modulators. A single ascending dose (SAD) portion of a phase I/II clinical study on the Moderna-partnered therapy is complete, while a multiple ascending dose (MAD) portion of the study is ongoing. Data from this Moderna-partnered MAD study is expected in the first half of 2025.

VRTX’s Zacks Ranks

Vertex currently carries a Zacks Rank #3 (Hold).

Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are ANI Pharmaceuticals (ANIP - Free Report) and 89bio (ETNB - Free Report) . While ANIP sports a Zacks Rank #1 (Strong Buy) at present, ETNB carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have risen from $5.54 to $6.35. EPS estimates for 2026 have increased from $6.75 to $7.21 during the same period. Year to date, shares of ANIP have risen 21%.

ANIP’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 17.32%.

Estimates for 89bio’s loss per share have narrowed from $2.51 to $1.98 for 2025 in the past 60 days. During the same time frame, estimates for 2026 loss per share have improved from $2.94 to $2.15. ETNB’s shares have lost 30% year to date.

89bio’s earnings missed estimates in three of the trailing four quarters and beat the mark on one occasion, delivering an average negative surprise of 46.18%.

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