I have a child with cystic fibrosis and would like to know if the new cystic fibrosis medication would work for her.
The FDA recently approved a new medication for the treatment of cystic fibrosis. The medication is called Trikafta and is actually a combination of three medications. The FDA approved this medication quickly using a priority review, because the medication was designated as Fast Track and Breakthrough Therapy. Even though the medication was approved more quickly than most, it was still rigorously examined for effectiveness and safety.
The medication is supposed to be used in individuals 12 years or older with a particular type of cystic fibrosis (at least one F508del mutation in the CFTR gene). Approximately 90 percent of individuals with cystic fibrosis carry this mutation and could potentially be treated with this new medication if they meet the age criteria. Clinical trials of this medication demonstrated that its use is associated with a significant improvement in breathing tests. The medication also caused improvements in sweat chloride (a test used in cystic fibrosis), fewer cases of worsening breathing symptoms and lung function, and an improvement in body mass index. This medication, like all medications, has some adverse effects but generally the therapy was well tolerated.
It would be well worth talking about this new medication with your health care provider. They can do the genetic test to determine if your child has the appropriate type of cystic fibrosis (if they have not already done this) and can walk you through the pros and cons of this new therapy versus the one that they are currently on.
John R. White is the chair of the Department of Pharmacotherapy at WSU-Spokane.