Kristi Rosa of RareDisease Report notes that over 70,000 are effected by cystic fibrosis and “although advances have been made in the fight against the progressive genetic disease which has allowed these patients to live longer lives, additional treatment options are needed.”
Rosa continued that two phase 3 trials will focus on “potential breakthrough therapy” with two compounds-VX-659 and VX-445–“which will be used as part of a triple combination modulator therapy for the disease.”
Children’s Hospital of Colorado and its Mike McMorris Cystic Fibrosis Research and Care Center will participate in one of the trials exploring the effectiveness of VX-659 in combination with tezacaftor/vacaftor (Symdeko) for CF patients 12 years of age and older who have 1 F508del mutation and a “minimal function” mutation. According to CHC “to date there is no CFTR modulator therapy available for CF patients with these mutation.”
The sponsor of VX-659 is Vertex.
vertex-initiates-first-phase- 3-vx-659-triple-combination- study
The sponsor of VX-445 is also Vertex
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