Therapy Areas: Hereditary Disorders
Vertex Pharmaceuticals selects VX-445 triple combination to submit for global regulatory approvals
3 June 2019 -

United States-based Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has chosen the triple combination of the new corrector VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential global regulatory approvals for patients aged 12 and older with cystic fibrosis, it was reported on Friday.

The basis of these submission will be the final data from a 24-week Phase three study in people with one F508del mutation and one minimal function mutation and from a four-week Phase three study in people with two F508del mutations. The company earlier revealed that both of these Phase three studies met its primary endpoints, and it revealed the final results of these studies, including results for key secondary endpoints and safety data.

The company plans to submit a New Drug Application to the United States Food and Drug Administration (FDA) in the third quarter of 2019 and a Marketing Authorisation Application to the European Medicines Agency in the fourth quarter of 2019 based on these data for patients with cystic fibrosis aged 12 years and older who have one F508del mutation and one minimal function mutation and in people with two F508del mutations.

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