Vertex Pharmaceuticals (VRTX) announced on Friday that the U.S. Food and Drug Administration (FDA) has broadened its approval of a cystic fibrosis medication to include additional patients with varying mutation types of the disease.
The FDA has sanctioned the extended use of Trikafta for cystic fibrosis (CF) patients aged 2 and above. Those eligible must possess at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a mutation that demonstrates a positive response to Trikafta according to clinical or laboratory data.
Notably, the safety profile regarding liver injury and liver failure has been updated from general warnings and precautions to a more serious boxed warning. Following this approval, 94 new non-F508del CFTR mutations have been included on the Trikafta label, thereby allowing approximately 300 more CF patients in the U.S. access to a treatment targeting the root cause of their condition for the first time.
Initially authorized in 2019, Trikafta has been a significant treatment option for tens of thousands of individuals with cystic fibrosis, as noted by Vertex's Chief Medical Officer, Dr. Carmen Bozic.
"Since its initial approval in 2019, TRIKAFTA has profoundly impacted the lives of countless individuals with cystic fibrosis," commented Dr. Carmen Bozic, Executive Vice President of Global Medicines Development and Medical Affairs, and Chief Medical Officer of Vertex. "With this latest approval, even more patients can now benefit from a treatment that addresses the root cause of their illness. We are committed to advancing efforts to expand the availability of our therapies to patients globally."
