Vertex Pharmaceuticals’ drug, Trikafta, is the first cystic fibrosis treatment designed to treat a mutation found in 90 percent of the U.S. cystic fibrosis population, or roughly 27,000 people.
Trikafta, which received orphan drug designation, is a combination of three drugs that target the defective cystic fibrosis transmembrane conductance regulator protein and help it function more effectively.
The FDA approved the treatment Oct. 21 for patients 12 and older who have at least one mutation in the CFTR gene.
Side effects of Trikafta include influenza, rash, headaches, upper respiratory tract infections and abdominal pains.
Read the full news release here.
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