FDA Approves New Drug for Most Common Form of Cystic Fibrosis

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A new drug to treat most cystic fibrosis patients has been approved by the FDA, with a list price of over $300,000 a year.

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Trikafta is the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Its list price is $311,000 a year, same as one of the maker's earlier treatments for the genetic disease.

Trikafta is approved for patients aged 12 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene. This includes about 90% of cystic fibrosis patients, or about 27,000 people in the United States, according to the FDA. There are about 2,000 known mutations of the CFTR gene, but the F508del mutation is the most common. Cystic fibrosis is the result of a defective protein caused by the gene mutation.

Current drugs that target the defective protein can be used to treat some patients, but many patients have mutations that don't respond to those treatments, the FDA said. Trikafta -- from Vertex Pharmaceuticals, Inc. -- is a combo of three drugs. It helps the defective protein made by the CFTR mutation function more effectively.

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That’s great!! So make medicine that could potentially make someone’s quality of life better but kill them with how much it cost... Pharmaceutical companies should be regulated with the astronomical amount they want to charge for medicine these days 😒

So umm 🤔 that’s just a little high

very cheap 🙃

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