Vertex Gets Expanded FDA Approval for Cystic Fibrosis Treatment

Dow Jones12-21 05:00

By Katherine Hamilton

Vertex Pharmaceuticals 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.

The medicine, called trikafta, can treat cystic fibrosis patients who have at least one F508del mutation in the transmembrane conductance regulator gene or a mutation that is responsive to trikafta based on clinical or in vitro data.

With this approval, 94 additional non-F508del CFTR mutations have been added to the trikafta label, making about 300 more people eligible for the treatment.

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.

Write to Katherine Hamilton at katherine.hamilton@wsj.com

(END) Dow Jones Newswires

December 20, 2024 16:00 ET (21:00 GMT)

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