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This new treatment helps 90% of cystic fibrosis patients breathe better

Vertex’s Trikafta was the second-fastest FDA approval in history, and could help some 27,000 patients.

In October 2019, Vertex Pharmaceuticals received FDA a­p­prov­al for Trikafta, a cystic fibrosis treatment that could help 90% of patients with the deadly genetic disorder. Cystic fibrosis, which affects about 30,000 people in the U.S. (and has a life expectancy of 44 years for patients born today), is caused by mutations in the CFTR gene that lead to a defective protein being produced. That protein doesn’t allow chloride to flow in and out of cells normally (particularly those that line the lungs) and prevents water from hydrating the cellular surface, leading to a buildup of thick mucus and difficulty breathing.

While other researchers looked to use gene therapy to address the disorder, Vertex’s team created a small-molecule drug that can tweak the faulty protein, allowing it to function more normally and drastically improving patient symptoms. “We were laughed at when we started,” says Vertex CEO Jeffrey Leiden. “No one thought that using small molecules would be enough to correct the underlying function of a giant protein.”

In trials of more than 400 patients, Trikafta increased lung function by 13.8% versus a placebo, decreased hospitalizations, and boosted quality of life. Based on strong clinical data, Trikafta was approved by the FDA in less than three months, the second-fastest approval in U.S. history.

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