Transcript for FDA approves new treatment for cystic fibrosis
Now to the breakthrough drug for cystic fibrosis. The fda approving a new treatment that could impact the lives of almost everyone living with the disease. Dr. Jennifer Ashton joins us with all the details on this news. So for people who are unfamiliar with cystic fibrosis, what are the basics of this rare disease. Michael, cystic fibrosis is a rare and potentially life-threatening disease that is Progressive. It's caused by a defect in a certain protein and leads to a buildup of mucus that affects almost organ system, respiratory, digestive, increasing the risk for infections and leading to diabetes. There is no cure so there's just treatment. And so this breakthrough with this new drug, we hear it's a little bit ahead of schedule. How is that possible and tell us a little more about it. That's part of the story behind the story. This drug got fda approval five months ahead of schedule which is definitely unusual. According to the fda, part of the reason for that it was granted priority review, fast track and breakthrough therapy status and orphan drug designation which is used to invent advise treatment for rare and life-threatening diseases, in terms of this drug it's called tricafta and thought to be a possible option for 90% of people suffering with cystic fibrosis who were previously out of options. It is approved for patients 12 years of age and older, clinical trials showed an improvement in lung function of about 10% to 13%. Some side effects, increase in liver function, flu and rash and the price tag for the drug, $311,000 a year. It remains to be seen how much insurance and medicaid will cover some or all of that. You know, when this happens, you get so excited about this breakthrough then you hear the price tag. Insurance companies will have to step up or it won't be available.
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