'Imagine waiting 28 years for a miracle—and then it suddenly happens.'
That's how Gunnar Esiason, a 28-year-old graduate student at Dartmouth College, described the approval of Trikafta, a new drug for cystic fibrosis.
That's why there was great excitement in the cystic fibrosis community when the U.S. Food and Drug Administration approved Trikafta on Monday, the first triple-combination therapy available to treat patients with the most common cystic fibrosis mutation. Its approval was fast-tracked via four different FDA programs that aim to provide treatments for rare or serious illnesses with unmet medical needs. This came after two clinical trials, the first of which showed improved measures of lung function in patients compared with a currently available drug. The second trial also showed improved lung function along and increased sweat production and body mass index in patients vs. a placebo.
Dr. Joan DeCelie-Germana, director of the Cystic Fibrosis Center at Northwell Health, has been treating patients with CF for over 30 years. In an interview with ABC News, she called the drug's approval"very exciting." Her center participated in the initial studies for Trikafta and also serves as the site of a current trial of the drug in patients 6 to 11.
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