Vertex' Trikafta Gets Expanded FDA Approval For Cystic Fibrosis Treatment

(RTTNews) - Vertex Pharmaceuticals (VRTX) said Friday the U.S. Food and Drug Administration expanded its approval of a cystic fibrosis treatment, encompassing more patients with different types of mutations of the disease.

FDA has approved the expanded use of Trikafta for the treatment of people with cystic fibrosis (CF) ages 2 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation that is responsive to Trikafta based on clinical and/or in vitro data.

In addition, safety information on liver injury and liver failure has been updated from warnings and precautions to a boxed warning. With this approval, 94 additional non-F508del CFTR mutations have been added to the Trikafta label, and approximately 300 additional people with CF in the U.S. are now eligible for a medicine to treat the underlying cause of their disease for the first time.

Trikafta was first approved in 2019. It has been used to treat tens of thousands of people with cystic fibrosis, said Vertex's chief medical officer, Carmen Bozic.

"Since its first approval in 2019, TRIKAFTA has had a transformative impact on tens of thousands of people living with cystic fibrosis," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. "With this approval, even more patients may be able to benefit from a medicine that treats the underlying cause of their disease, and we look forward to continuing the work to extend the approvals and availability of our medicines to patients around the world."