New Cystic Fibrosis Drug Reduces Treatment Burden

Every day, tens of thousands of people with cystic fibrosis follow a strict routine, taking multiple daily medications and receiving respiratory treatments. This daily regimen is ingrained into their lives, but a new drug approved by the U.S. Food and Drug Administration (FDA) may help to decrease some of that treatment burden. Children’s Hospital Colorado pulmonologist Jordana Hoppe, MD, is at the forefront of this treatment breakthrough, transforming cystic fibrosis care nationwide.

In December 2024, the FDA approved Alyftrek for individuals with cystic fibrosis. This once-daily CFTR modulator is now available for approximately 90% of people with cystic fibrosis 6 years and older, based on their genetic variants.

Cystic fibrosis (CF) is a genetic disorder that causes severe damage to the lungs, digestive system and other organs. It’s caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein produces thick and sticky mucus in various organs, which can block airways and passageways and cause severe complications. CFTR-modulator drugs, such as Alyftrek, target the defective protein to help improve lung function, lower the risk of respiratory infections, and reduce symptoms.

Dr. Hoppe led a pediatric study evaluating the safety and efficacy of Alyftrek. The study enrolled 78 children with CF. Before participating in the trial, they were required to be on Trikafta, a current CFTR modulator, for at least four weeks. The participants had baseline assessments before switching to the new medication.

Researchers determined that the new drug is safe for the pediatric population, with most adverse events being mild to moderate in severity and often similar to symptoms of cystic fibrosis.

Not only is the medication safe, but it’s effective too. Participants experienced an average decrease of eight points in sweat chloride values, the measurement used to diagnose cystic fibrosis based on the restoration of CFTR function. Through 24 weeks of treatment with Alyftrek, 95% of the participants recorded sweat chloride values lower than the diagnostic threshold for cystic fibrosis (<60mmol/L) and 53% recorded sweat chloride values in the normal range (<30mmol/L).

“To say someone has cystic fibrosis, they have to have a sweat chloride test of 60 or higher,” Dr. Hoppe explains. “The percentage of participants that had a sweat chloride below 60, the diagnostic criteria for CF, and below 30, a sweat chloride value that is in the normal range, is higher than the clinical trials of Trikafta.”

The international pediatric study ran concurrent with the adolescent and adult studies, allowing a younger group of patients to have earlier access to this new treatment.

Over the past few decades, cystic fibrosis treatments and life expectancy have improved significantly, thanks to advancements like CFTR modulators. These drugs are absorbed best when taken with a high-fat meal, and eating two high-fat meals daily, as required for Trikafta, can be challenging. Alyftrek helps ease some of the treatment burden for people with CF and their families.

“Anytime we can decrease the amount of medicine someone has to take, it’s a good thing,” Dr. Hoppe says.

Even with these incredible advances, Dr. Hoppe says there’s still more work to do. Around 10% of people with CF are not eligible for CFTR modulator therapy due to their genetic variants. Researchers are committed to exploring new treatment approaches to enhance the health and quality of life for everyone affected.