Newly-approved drug called ‘significant breakthrough’ for cystic fibrosis

Simple Health

AUSTIN (KXAN) — The FDA has approved a new drug for the treatment of cystic fibrosis (CF), a medicine people in the pharmaceutical community are calling a “significant breakthrough.”

The drug, TRIKAFTA, was approved for people ages 12 years and older and is based on two positive global Phase 3 studies. Doctors say this has the potential to treat up to 90% of all people with CF in the future.

The FDA approved TRIKAFTA on Monday for the treatment of of cystic fibrosis. (Courtesy: Carolyn Heinerichs)

“[This] is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” said Steven Rowe, M.D., the director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama at Birmingham.

CF is a rare genetic disease known to shorten lifespans and impacts internal organs including the lungs, liver, sinuses, sweat glands, pancreas and reproductive tract, experts say. It affects 75,000 people worldwide with a median age of death in the early 30’s.

Patient success

Dr. Bennie McWilliams, the director of the Cystic Fibrosis Center in Austin said patients typically spend 15 to 20 hours each week dealing with CF medications, treatments and therapies. He monitors the health of the same patients throughout the course of their lives at the CF clinic at Dell Children’s.

“It’s about the equivalent of having a part time job just for taking care of your disease,” Dr. McWilliams said.

TRIKAFTA is a combination of medications based on decades of research. Dr. McWilliams said previous drugs wouldn’t treat the underlying protein deficiency found in the disease.

“We were able to see a greater improvement in patients,” Dr. McWilliams told KXAN. “They were gaining weight, had more energy, feeling better and breathing easier.”

Mac Rung, a 19-year-old University of Texas freshman, was a participant in the clinical trials. He’s been taking TRIKAFTA for the past year. He said he has seen significant improvements which has given him new hope.

“This drug will save lives,” Rung said. “Within two weeks, my lung function jumped up around 10 percent which is not something you usually see, especially in a span of two weeks.”

Rung said he takes three TRIKAFTA pills per day with food: two in the morning and one in the evening. He called the routine “manageable.”

He said the medication not only will improve the health of those living with cystic fibrosis, but will greatly impact their quality of life.

“People like me will be able to go to college and get their degree without being held back by cystic fibrosis. And that’s something that would not have happened 10 to 15 years ago,” Rung said.

Hefty price tag

Early numbers suggest that TRIKAFTA will carry a large cost. Dr. McWilliams said the previous medications have historically hovered in the hundreds of thousands per year of treatment. Rung said TRIKAFTA is being offered for $311,000 per year.

Dr. McWilliams said insurance companies should be willing to help out. He said the quality of the research will make it easy to present its effectiveness to pharmaceutical companies.

“These are not just new designer drugs, but really something that will have a huge impact on lives,” Dr. McWilliams said. “We’re giving these patients the opportunities to do all the things that everyone wants to do.”

On KXAN News at 9 and 10 p.m., Alex Caprariello will interview Mac Rung, a UT freshman who was on the clinical trials for TRIKAFTA. He’ll share the big improvements he felt through its use.

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