FDA批准kaftrio(Trikafta)用于治疗2岁及以上患有囊性纤维化(CF)的患者

On December 20, 2024, Vertex announced that the U.S. Food and Drug Administration (FDA) has approved the expansion of the use of Trikafta to treat patients 2 years and older with cystic fibrosis (CF).

They have at least one F508del mutation, located in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a mutation that responds to Trikafta based on clinical and/or in vitro data. The safety information regarding liver injury and liver failure has been updated from a warning and precautionary measure to a black box warning. With this approval, 94 new non-F508delCFTR mutations have been added to Trikafta's label.

On December 20, 2024, Vertex (Vertex Pharmaceuticals) announced that the U.S. FDA has approved the indication expansion of its triple combination therapy Trikafta: it can now be used to treat cystic fibrosis (CF) patients 2 years old and older who carry at least one F508del mutation or 94 newly incorporated response mutations. This landmark approval is based on clinical and in vitro data verification and will give approximately 300 CF patients in the United States who have no previous targeted treatment options the opportunity to treat their cause for the first time.

On April 26, 2023, Vertex announced that the U.S. Food and Drug Administration (FDA) has approved the expansion of the applicable population of Trikafta to include cystic fibrosis (CF) pediatric patients aged 2 to 5 years old. These patients need to carry at least one F508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a CFTR gene mutation based on in vitro data showing a response to Trikafta. Previously, the FDA had approved Trikafta for cystic fibrosis patients 6 years of age and older who carry at least one F508del mutation or a CFTR gene mutation that shows a response to it based on in vitro data.

On June 9, 2021, Vertex announced that the U.S. Food and Drug Administration (FDA) has approved an expanded applicable population to include children with cystic fibrosis (CF) aged 6 to 11 years old. These patients need to have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a CFTR gene mutation that shows response to Trikafta based on in vitro data.

On December 21, 2020, Vertex announced that the U.S. Food and Drug Administration (FDA) has expanded the scope of Trikafta's applicable population to include cystic fibrosis (CF) patients 12 years old and above. These patients need to carry certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that have been confirmed by in vitro data to be responsive to Trikafta.

On October 21, 2019, Vertex announced that the U.S. Food and Drug Administration (FDA) has approved Trikafta for the treatment of cystic fibrosis (CF) patients 12 years of age and older who carry at least one F508del mutation (the most common cystic fibrosis-causing mutation) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease that affects more than 92,000 people worldwide. Cystic fibrosis is a progressive, multi-organ disease that affects the lungs, liver, pancreas, gastrointestinal tract, sinuses, sweat glands, and reproductive system.

Cystic fibrosis is caused by defects and/or loss of CFTR protein caused by certain mutations in the CFTR gene. A child must inherit two defective CFTR genes (one from each parent) to develop cystic fibrosis, and these mutations can be identified through genetic testing.

Although there are many different types of CFTR mutations that cause the disease, the vast majority of cystic fibrosis patients carry at least one F508del mutation. CFTR mutations cause cystic fibrosis by making the CFTR protein defective or leading to insufficient or missing CFTR protein on the cell surface. Functional defects and/or loss of the CFTR protein lead to poor flow of salt and water inside and outside cells in multiple organs.

In the lungs, this causes a buildup of abnormally thick, sticky mucus, causing chronic lung infections and progressive lung damage, ultimately leading to death in many patients. The median age of death among patients is in their 30s, but with treatment, survival expectations are improving.

Today, Vertex's cystic fibrosis treatments are treating more than 68,000 cystic fibrosis patients in more than 60 countries on six continents. This represents two-thirds of patients with confirmed cystic fibrosis who are eligible for treatment with CFTR modulators.