As reported in Cystic Fibrosis News Today, Vertex is running a phase 3 trial with up to 82 investigative sites with Children’s Hospital Colorado’s Mike Morris Cystic Fibrosis Research and Care Center playing a key role in supporting the study. As reported by Alice Melao, the phase 3 study “is assessing the efficacy of VX-659 in a triple combination with symdeko,(tezacftor/ivacaftor and ivacaftor).” The trial is listed at clinicaltrials.gov https://clinicaltrials.gov/ct2/show/NCT03447249?term=VX-659&cond=Cystic+Fibrosis&rank=2. “will test the triple combination of cystic fibrosis (CF) patients with one F508del mutation and one minimal function mutation in the CFTR gene (the gene that is defective in CF patients).
According to the Cystic Fibrosis Foundation more than 30,000 people live with cystic fibrosis in the United States and more than 70,000 worldwide. There are approximately 1,000 new cases diagnosed each year. Approximately 75% plus of people with CF are diagnosed by 2.
TrialSite News staff looked into the costs for treating CF. On average CF treatment cost in the US is $15,571 and mild, moderate and severe disease cost ranges from $10,151, $25,647 and $33,691. Lifetime health costs are approximately $306,332. https://www.sciencedirect.com/science/article/pii/S1098301512042684
Vertex’s first CF drug Kalydeco generated $845 million in sales in 2017 according to FactSet Systems. Orkambi another Vertex CF drug generated $1.32 billion in sales in 2017.
In addition to Children’s Hospital of Colorado other research sites participating in the Vertex study include:
University of Alabama, Stanford, Harford Health, Yale New Haven Medical Center, University of Miami, Miller School of Medicine, Nicklaus Children’s Hospital (Miami), Arnold Palmer Hospital (Orlando), Johns Hopkins All Children’s Hospital Outpatient Care Center (Saint Petersburg, FL), St. Luke’s CF Center Idaho, Cystic Fibrosis Center Chicago and dozens of other major academic centers).
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