FDA Approves 'Breakthrough' Cystic Fibrosis Drug Trikafta

The U.S. Food and Drug Administration (FDA) approved a new “breakthrough” therapy for patients with cystic fibrosis over the age of 12, which may impact up to 90% of cystic fibrosis patients.

On Monday, the FDA announced it gave approval to a new triple-combination drug therapy, brand name Trikafta (elexacaftor/ivacaftor/tezacaftor), for the treatment of cystic fibrosis. Cystic fibrosis (CF) is a rare, progressive genetic condition that primarily affects the lungs and digestive track, causing thick mucus to develop, which can lead to life-threatening infections, inflammation and lung and respiratory issues. CF impacts approximately 27,000 people in the United States, according to the FDA.

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which prevents the production of CFTR proteins and inhibits how much fluid and salt moves in and out of your cells, according to the Cystic Fibrosis Foundation. As a result, mucus builds up in the lungs and blocks airways and traps bacteria, causing infection. Mucus that collects in the pancreas prevents key nutrients from being absorbed.

Trikafta targets CFTR proteins affected by gene mutations to help them function more efficiently in the body. Unlike other available drugs that target more limited gene mutations, Trikafta can be used by patients who have at least one F508del mutation in their genes. This is the most common mutation (out of 2,000 known CFTR mutations) seen in up to 90% of patients with cystic fibrosis.

Trikafta, which was developed by Vertex Pharmaceuticals Incorporated, was fast-tracked by the FDA and reviewed and approved in about three months, the FDA said. Vertex submitted two clinical trials that showed Trikafta improved lung function more effectively than a non-active placebo or other, more limited available CFTR-targeting drugs. One of the trials also showed an improvement in sweat production and body mass index.

In general, the FDA decided Trikafta was relatively safe. The most common side effects included rashes, flu symptoms, headaches, upper respiratory tract infections, abdominal pain, diarrhea, nasal congestion, mucus in the nasal cavity and sinus infections. Trikafta may increase elevated liver function, so the FDA said patients need to take the drug with others that reduce these liver enzymes.

“Today’s landmark approval [makes] a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” said acting FDA Commissioner Ned Sharpless, M.D., in a press release, adding:

In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That’s why we used all available programs, including Priority Review, Fast Track, Breakthrough Therapy, and orphan drug designation, to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards.

The approval of the triple-combination modulator represents a truly transformational milestone in the fight against #cysticfibrosis. We could not be more excited about this pivotal moment in the history of CF. https://t.co/OEVnfRIaKc

— Cystic Fibrosis Foundation (@CF_Foundation) October 21, 2019

Trikafta has been hailed as an “exciting” “miracle” drug by the cystic fibrosis community, including Dr. Joan DeCelie-Germana, director of the Cystic Fibrosis Center at Northwell Health, whose treatment center participated in clinical trials of Trikafta.

“Patients had an improvement in lung function and less illness [and] they will enjoy more days without antibiotics and better quality of life,” DeCelie-Germana told ABC News. “It’s inspirational. I couldn’t have even thought of this 30 years ago. It’s not a cure, but it’s the closest thing we have.”

According to ABC News, Vertex will make Trikafta available within two weeks “through speciality pharmacies.” It will carry an average annual cost of $311,000, which Vertex representatives told ABC is likely to be “reimbursed by private insurers and Medicaid/Medicare.”