Oct 21, 2019

FDA approves drug therapy that would treat 90% of people with cystic fibrosis

Mucoviscidosis (accumulation of mucus in the respiratory tract), seen on a frontal X-ray of the chest. Photo: BSIP/Universal Images Group via Getty Images

The Food and Drug Administration has approved a triple combination therapy manufactured by Vertex that would treat 90% of patients with cystic fibrosis.

The big picture: The approval of Trikafta came in record time at five months and will be priced at $311,503 annually, or $23,896 per 28-day pack, according to the Securities and Exchange Commission.

  • Cystic fibrosis is a chronic, progressive and frequently fatal genetic disease that primarily affects the respiratory and digestive systems of children and young adults.
  • Patients over the age of 12 with at least one F508del mutation of the inherited disease will be eligible for the therapy. In clinical trials, patients who received Trikafta experienced significant improvements in lung function, STAT News reports.
  • Vertex shares jumped more than 4% on the news.

What they're saying:

“In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That’s why we used all available programs ... to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards."
— Acting FDA commissioner Ned Sharpless

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