CORPUS CHRISTI, Texas — Dr. Salim Surani called Trikafta a 'game-changer' for the treatment of cystic fibrosis. The debilitating lung and GI disease is considered rare, progressive and life threatening by the FDA and medical professionals.
Trikafta is the first, triple combination therapy that has been approved by the FDA for patients who are 12 years old and up. The FDA said the treatment can be given nearly 90 percent of cystic fibrosis patients who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator. Trikafta was originally going to be reviewed in March 2020.
"So they felt that this was so important for the patient and it's such a rare disease they actually lived to their promise and they approved the drug much earlier," Dr. Surani said.
Years ago, Dr. Surani said CF was considered to be a childhood disease because few patients lived to see adulthood but now, they're living longer and with a better quality of life. Surani said this means, doctors worldwide are looking into more ways to treat adults with the disease.
"I think that is a game-changer for not only physicians for research, scientists, and the FDA because they consider that this is a rare disease," he said.
The subject is near and dear to the doctor's heart. In 1996, Dr. Surani performed his first lung transplant; the patient had cystic fibrosis.
"It still echos in my mind everyday," he said.
Back then, Dr. Surani said only 1000 lung transplants had been completed worldwide and the mortality rate was 50 percent. Even knowing her odds, Surani's patient went through with the surgery.
"She is one of the people who helped shape me, who I am, she said 'Dr. Surani, the moment I took the first breath after transplant that was such a nice breath that one breath was worth all the pain going,'" he said.
Unfortunately, she died 11 months after the transplant.
According to a release from the FDA, Trikafta has not been approved for patients who are younger than 12 years old.
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