Cystic fibrosis patients, doctors celebrate FDA's 'life-changing' approval of new treatment

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FDA approves drug combination that offers new hope for cystic fibrosis patients

25-year-old Laney Siems is slated to try new treatment which targets the defective protein responsible for cystic fibrosis and causes a build-up of mucus in the lungs.

The FDA’s recent approval of a three-drug combination therapy for cystic fibrosis patients is being celebrated among both patients and doctors as “life-changing” news. Trikafta, produced by Vertex Pharmaceuticals Inc., targets the defective protein responsible for the disease, which causes a build-up of thick mucus in the lungs and other organs.

It’s the first triple combination therapy available to treat those with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is the most common among patients. Among those eligible to receive Trikafta is Gunnar Esiason, son of former NFL quarterback Boomer Esiason, who began the treatment in a clinical trial last April.

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“My life changed on April 9, 2018 when I took my first dose of the ‘Triple Combo’ in a phase III clinical trial,” Gunnar Esiason tweeted on Oct. 21, the day the FDA announced its decision. “The triple combo, named for its various components, is a drug designed to treat the underlying cause of cystic fibrosis. There was a 50/50 shot I would get the active drug or the placebo, but I knew I had the real thing because it worked in a matter of hours. Since then, everything has changed.”

Gunnar Esiason said the health agency’s decision will put the treatment in the hands of more patients, and that he “cannot wait to see how people with CF will work to change the world now that nearly our entire community will have access to this life-changing medication.”

The treatment, while not a cure, is for patients who have at least one copy of the F508del mutation and involves taking several pills each day. According to Gunnar Esiason, it has helped him “pursue a life largely independent of the harsh symptoms that come with cystic fibrosis.”

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In clinical trials, patients with two copies of the F508del mutation had a 10 percent increase in lung function, and people with one copy had more than a 14 percent increase compared to placebo.

“The drug has given me the life that was stolen from me as I creeped towards end-stage illness,” he tweeted.

The Cystic Fibrosis Foundation also called the FDA’s approval a “tremendous breakthrough” for patients with the disease. More than 30,000 people are living with cystic fibrosis in the U.S., with approximately 1,000 new cases diagnosed each year, according to the CF Foundation. More than half of the patient population is age 18 or older, and it is believed that the drug could become available to more than 90 percent of patients.

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“As we celebrate today, we will not lose sight of the many individuals in our community who are still waiting for a breakthrough that will treat their mutations,” said Michael P. Boyle, M.D., senior vice president of therapeutics development for the CF Foundation. “We will not be done until every person with CF has a treatment for the underlying cause of their disease and, one day, a cure.”