HQ Team
December 21, 2024: Vertex Pharmaceuticals Inc.’s Alyftrek drug got clearance from the Food and Drug Administration to expand its treatment for cystic fibrosis in people six years and older, according to a statement from the company.
The regulator’s approval was based on an end-stage trial on more than 1,000 patients across more than 20 countries and more than 200 sites, according to the Boston, US-based company’s statement.
“In the phase 3 study of children with cystic fibrosis ages 6-11 years, Alyftrek demonstrated safety, the primary endpoint.” Secondary endpoints also supported the benefit of the drug in this age group.
The FDA, in 2019, approved the company’s drug Trikafta to treat patients with cystic fibrosis who are more than 12 years old.
Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. It affects more than 92,000 people globally.
It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene.
While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation. The median age of death is in the 30s, but with treatment, projected survival is improving.
Trikafta is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively.
“Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function — Alyftrek, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than Trikafta, is another step in achieving this goal,” Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
Alyftrek was also submitted to global health authorities and is under regulatory review in the European Union, the United Kingdom, Canada, Switzerland, Australia and New Zealand.
