Vertex’s triple combo cystic-fibrosis drug meets main late-stage study goals, ET HealthWorld


Bengaluru: Vertex Pharmaceuticals‘ experimental cystic-fibrosis treatment met all of its main goals in a late-stage study when tested in patients aged 12 years and older, the company said on Monday.

The once-daily triple combination treatment, called vanza triple, met the primary goals of the study’s two 52-week trials and was non-inferior for lung function to Trikafta, the company’s top-selling drug for the disorder, with a comparable safety profile.

A third 24-week trial, testing the drug in children who were six to 11 year old, demonstrated the potential of keeping the disease in check by treating it early in life.

Vertex plans to file for approval with global regulators for patients who are six years and older by mid-2024 and may use a priority review voucher in the United States

The company also forecast 2024 sales above Wall Street estimates on Monday, banking on expectations for its CF treatments and the launch of gene therapy Casgevy.

For 2024, it expects revenue of between $10.55 billion and $10.75 billion, above average analyst estimates of $10.61 billion, according to LSEG data.

Trikafta sales jumped 15.4 per cent to $2.33 billion, compared with estimates of $2.31 billion

On an adjusted basis, Vertex earned $4.20 per share for the quarter ended Dec. 31, ahead of analysts’ average estimate of $4.10 per share.

CF – an inherited disorder that causes severe damage to the lungs, digestive system and other organs – affects an estimated 105,000 people across 94 countries, according to data from U.S.-based CF Foundation.

Shares of the Boston Massachusetts-based company were up 2.8 per cent in trading after the bell.

  • Published On Feb 6, 2024 at 02:39 PM IST

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