FDA Approves 'Miracle' Drug Expected To Treat 90% Of Cystic Fibrosis Patients

A new breakthrough drug to treat the most common form of cystic fibrosis in patients has officially been approved by the U.S. Food and Drug Administration, Forbes reported.

Trikafta is the first triple combination therapy available to treat CF patients and has already been dubbed a 'miracle' treatment by one of its trial users.

Cystic fibrosis is a progressive genetic disorder that largely affects the lungs, but can also affect the pancreas, liver, kidneys, and intestine.

YouTube | Cystic Fibrosis Foundation

According to the Cystic Fibrosis Foundation, patients suffer from a build up of mucus in various organs, which in the case of the lungs can lead to infections, inflammation, respiratory failure, and other serious complications.

More than 70,000 people are living with CF worldwide, and approximately 1,000 new cases are diagnosed each year.

Some 90 percent of those with CF have at least the 1 F508del mutation which is the cause for their illness.


But the newly approved Trikafta drug therapy has proven to be able to treat those patients who are suffering from the most common cystic fibrosis mutation.

According to ABC News, 28-year-old former NFL quarterback Gunnar Esiason has been living with CF since 1993 and saw his symptoms significantly worsen in 2013.

In 2018, he enrolled in a clinical trial for Trikafta, which is when he said his life completely changed.

As he explained, "Imagine waiting 28 years for a miracle, and then it suddenly happens."

Unsplash | Nghia Le

Within the first few days of treatment, Esiason noticed an improvement in his breathing and appetite. Within the first few weeks he gained weight, and his pulmonary function tests improved.

The FDA has approved the medication for patients 12-years-old and older.

The Cystic Fibrosis Foundation

Trikafta was fast-tracked by four different FDA programs after two clinical trials were completed, the first with 403 patients and the second with 107. Both trials showed significant improvement in lung function among patients.

There were some adverse reactions too, including headaches, upper respiratory tract infections, abdominal pain, diarrhea, rashes, and increased liver enzymes.

Trikafta works by helping defective CFTR protein in patients with 1 or 2 F508del mutations function more effectively.


In most patients, this protein is what results in the excess mucus in their lungs, leading to breathing obstruction, infection, and in some cases, death.

Dr. Joan DeCelie-Germana, director of the CF Center at Northwell Health, said the drug's approval was "very exciting."

Unsplash | Nine Köpfer

She told ABC News that patients who took part in the treatment's trials "had an improvement in lung function and less illness," adding that "they will enjoy more days without antibiotics and better quality of life."

"It's inspirational," she said. "I couldn't have even thought of this 30 years ago. It's not a cure, but it's the closest thing we have."

h/t: Forbes, ABC News

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