US FDA approves Vertex Pharma's triple combo cystic fibrosis treatment

Reuters
2024.12.20 21:00
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The U.S. FDA has approved Vertex Pharmaceuticals' new triple combination treatment for cystic fibrosis, branded Alyftrek, for patients aged 6 and older. This approval enhances Vertex's position in the cystic fibrosis market, where it has already made significant advancements with its previous drug, Trikafta. Cystic fibrosis affects around 40,000 individuals in the U.S., leading to severe respiratory issues. The company has not yet disclosed pricing or availability details for Alyftrek.

Dec 20 (Reuters) - The U.S. Food and Drug Administration has approved Vertex Pharmaceuticals’ (VRTX.O) next-generation treatment for a rare and progressive genetic disease, the company said on Friday, expanding its dominance in the market for cystic fibrosis.

The drugmaker, with almost $10 billion in sales last year, has significantly impacted cystic fibrosis treatment, transforming it from a disease with a median death age of around 30 years to a manageable condition through its drugs — such as its top-selling Trikafta.

The once-daily triple combination therapy, branded Alyftrek, was approved to treat patients aged 6 years and older.

Cystic fibrosis is an inherited disease in which the absence of a protein results in poor flow of salt and water into and out of the cells in a number of organs.

In the lungs, it causes sticky, thick mucus to build up, making it difficult to breathe and leading to progressive lung damage that can eventually result in death.

There are about 40,000 people with cystic fibrosis in the United States, according to the American Lung Association.

The company did not immediately respond to a Reuters request on pricing and availability.