University of Kansas Health System Contributes to the Successful Phase III Cystic Fibrosis Treatment now FDA Approved

Nov 2, 2019 | CF, Cystic Fibrosis, University of Kansas Health System, Vertex Pharmaceuticals

University of Kansas Health System Contributes to the Successful Phase III Cystic Fibrosis Treatment now FDA Approved

The University of Kansas Health System is one 115 global sites participating in a worldwide study for a new treatment considered by some as a “game-changer.” Focusing on a new therapy for cystic fibrosis, the new treatment was invented in part by the Cystic Fibrosis Foundation. The study is having a direct impact on patients.

A Strong Statement

Steven Stites, MD, Chief Medical Officer of the University of Kansas Health System noted “I’ve seen a lot of patients die from this disease. I’ve been doing it for a long time.” He declared powerfully “I’ve never seen anything like this” reported Fox4 Kansas City.

A Patient’s Story

Sarah Carollo has been participating in this cystic fibrosis study. Part of the trial group, she has been taking the drug since last summer reported the Fox news outlet. The treatment looks incredibly promising. Ms. Carollo notes “More than anything, I noticed that I could walk as fast as others” and, “I noticed I could pick up and move things at my work. Just an overall quality of life increase is just remarkable.”

And Sarah is taking advantage of this progress—participating in a 5K race and more active enjoying simple but powerful things like being able to move the body with more ease—the freedom to live become truly appreciated when a medical breakthrough offers a second chance. It feels like a miracle.

Cystic Fibrosis Past, Present & Future

Although CF used to take the lives of teens and young adults, a number of new therapies have made it more manageable than in years past. University of Kansas Health System pulmonologist Deepika Polineni noted “There are more people living with CF as adults than there are children. That’s because of the improved survival overall.”

The physicians presented their findings to the New England Journal of Medicine this past Thursday.

The Treatment & Access

Physicians report the treatment may be effective for up to 90% of the population living with CF. The price tag isn’t cheap at $300,000 per annum. Approval by Payers (public sector ones including Medicare and Medicaid on a state by state basis) and of course via private insurers and self-insured companies.

The Drug: Trifafta

The FDA recently approved Vertex Pharmaceuticals Trikafta. TrialSite News recently reviewed FDA approval

The Study

The study evaluated the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects). The study included the participation of 115 clinical investigational sites.

Call to Action: If you or a loved one have a cystic fibrosis diagnosis consider asking your physician to review this study.

Source: Fox4 TV

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