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A Second Breath for Cystic Fibrosis Patients

Oct 21, 2019 by Esteban Kosak

A new treatment for cystic fibrosis promises remarkable improvement in the quality of life for patients who suffer this illness as the FDA approves Trikafta.

Cystic fibrosis is a rare, chronic, life-threatening disease that results from the mutation of the CFTR gene, which alters the production and synthesis of proteins. There are almost 2,000 known mutations to the CFTR gene, and the F508del mutation is the most common one. Defective CFTR proteins cause the production of thick mucus that build-ups in the respiratory and digestive systems, leading to severe consequences and complications such as infections and diabetes.

New treatment for cyctic fibrosis
Photo credit: Polina Tankilevitch from Pexels

Trikafta is a combination of three different drugs (elexacafor/ivacaftor/tazacaftor) that target defective CFTR proteins and make them more functional. The groundbreaking innovation with this newly approved drug is that it is the first effective treatment for patients with cystic fibrosis that are 12 years and older that possesses at least one F508del mutation. 90% of the cystic fibrosis patient population could be treatable as it is the most common mutation!

Two trials published by The Lancet and the New England Journal of Medicine, have demonstrated the efficacy and safety of Trikafta. Patients on the Trikafta group showed an increased mean percent of the predicted forced expiratory volume in one second or ppFEV1 (a marker used in cystic fibrosis lung disease progression) by 10-13% compared to the placebo group baseline. Even more, the first trial treatment with Trikafta also showed improvements in sweat chloride concentration, body mass index, and a reduced risk of respiratory infections.

"Today's landmark approval is a testament to these efforts, making 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 additional effective therapy," said acting FDA Commissioner Ned Sharpless, M.D.

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