Vertex Highlights First Oral Presentation of Phase 3 Clinical Dataof the Vanzacaftor Triple Combination and New Data on Long-Term Impact of TRIKAFTA at the North American CysticFibrosis Conference
Natalia Elliot | September 30, 2024 | News story | | Vertex Pharma
- Phase 3 data on investigational vanzacaftor triple combination demonstrates noninferiority
to TRIKAFTA® in ppFEV1 and further improvement of CFTR function
as measured by sweat chloride – - Real-world evidence and clinical studies of TRIKAFTA® continue to show
sustained long- term benefits including improvement in pancreatic function
in young patients
September 26 2024 – Boston, US – Vertex Pharmaceuticals Incorporated (Nasdaq:
VRTX) today announced the first accepted medical presentations of the Phase 3 data on
the investigational once daily vanzacaftor/tezacaftor/deutivacaftor (“vanzacaftor triple
combination”) — the potential next-in-class triple combination medicine — will take
place at the North American Cystic Fibrosis Conference (NACFC). Vertex also announced
presentations describing long- term outcomes in people with cystic fibrosis (CF) ages 2 to
11 years taking TRIKAFTA®, also known in the European Union and in the U.K. as
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor,
demonstrating consistent and sustained improvements across multiple measures of
disease.
Data on the Investigational Once Daily Next-in-Class Triple Combination Therapy,
vanzacaftor/tezacaftor/deutivacaftor
Vertex will present data on the vanzacaftor triple combination in an oral presentation and
two posters on Friday, September 27. This is the first time that the clinical data from the
Phase 3 clinical trials of the vanzacaftor triple combination in patients 6 years and older
with CF have been accepted for presentation at a medical meeting. These data formed the
basis of global regulatory submissions.
Data from the Phase 3 clinical studies of the vanzacaftor triple combination will be
presented in the following sessions:
- “Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Adolescents and Adults with
- CF: Results from Two Randomized, Active-Controlled Phase 3 Trials,” will be an
- oral presentation during a session entitled: “Cutting Edge Research: What’s New” on
- Friday, September 27, from 10:15 a.m. to 12:15 p.m. EDT and be a poster presentation
- on Friday, September 27, from 1:15-2:15 p.m. EDT.
“Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) inChildren 6 Through 11 Years of Age with Cystic Fibrosis,” will be a poster presentation on Friday, September 27, from 1:15-2:15 p.m. EDT.
The vanzacaftor triple combination was granted Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for the treatment of CF and has been submitted for regulatory approval in the U.S., Canada, U.K., EU, Switzerland, Australia and New Zealand. Vertex has been assigned a Prescription Drug User Fee Act (PDUFA) date by the FDA of January 2, 2025, for this submission. The vanzacaftor triple combination has not been approved by any global health authority.
About Vertex: Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases – cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia – and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 14 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
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