Therapy Areas: Hereditary Disorders
Vertex Begins Phase 3 Studies of Triple Combination Regimen for People with Cystic Fibrosis
1 May 2018 - - Boston, Massachusetts-based serious disease therapeutics developer Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is initiating two Phase 3 studies of VX-445, tezacaftor, and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF), the company said.
The first Phase 3 study will evaluate approximately 360 people with CF who have one copy of the F508del mutation and one minimal function mutation, and is designed to support the submission of a new drug application in the US using data from the study's 4-week primary efficacy endpoint together with safety data through 12 weeks of treatment.
The second Phase 3 study will evaluate approximately 100 people with CF who have two copies of the F508del mutation, the most common genetic form of the disease, and is designed to support the submission of an application for approval in patients with two copies of the F508del mutation in the US using data from the study's 4-week primary efficacy endpoint together with 24-week safety data generated from the Phase 3 study in patients with one F508del mutation and one minimal function mutation.
Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.
In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
The company has research and development sites and commercial offices in the US, Europe, Canada, and Australia.
Login
Username:

Password:


Related Headlines