Health and Science

FDA approves Vertex Pharma's combo treatment for cystic fibrosis

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Key Points
  • The U.S. Food and Drug Administration said on Monday it had approved Vertex Pharmaceutical's three-drug combination for cystic fibrosis.
  • The approval comes months ahead of the FDA's previously announced action date in March, sending the drugmaker's shares up 4.1% at $183.53 in late afternoon trading.
  • CF is a chronic, progressive, and frequently fatal genetic disease, primarily affecting the respiratory and digestive systems in children and young adults.
The U.S. Food and Drug Administration
Jacquelyn Martin | AP

The U.S. Food and Drug Administration said on Monday it had approved Vertex Pharmaceutical's three-drug combination for cystic fibrosis (CF), a rare genetic condition.

The approval comes months ahead of the FDA's previously announced action date in March, sending the drugmaker's shares up 4.1% at $183.53 in late afternoon trading, before being halted for news pending.

The treatment, Trikafta, is a combination of three drugs that target a defective protein responsible for the life-threatening disease that causes the build up of thick mucus in body parts such as the lungs and digestive tract.

The backbone drugs in Trikafta are ivacaftor and tezacaftor, which make up Vertex's other approved treatment for CF called Symdeko.

CF is a chronic, progressive, and frequently fatal genetic disease, primarily affecting the respiratory and digestive systems in children and young adults.

Trikafta is the first approved treatment that is effective for patients 12 years and older, who have a genetic mutation which affects 90% of CF patients or roughly 27,000 people in the United States, the agency said.