Business Wire

Vertex Announces Reimbursement Agreement in Australia for ORKAMBI® (lumacaftor/ivacaftor) for People with Cystic Fibrosis Ages Six Years and Older with Two Copies of the F508del Mutation

Del

Vertex Pharmaceuticals (Europe) Limited announces finalization of an agreement with the Australian Government that allows for reimbursement of ORKAMBI® (lumacaftor/ivacaftor) for people ages six and over with cystic fibrosis (CF) who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This agreement means lumacaftor/ivacaftor will be listed on the Pharmaceutical Benefits Scheme (PBS) from 1 October and follows a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC). A pathway to access for future Vertex CF medicine, tezacaftor/ivacaftor, has been established as part of this process.

“We are delighted that lumacaftor/ivacaftor will be made available to eligible CF patients in Australia,” said Stuart Arbuckle, Executive Vice President and Chief Commercial Officer at Vertex. “We would like to recognize the PBAC for seeing the value of this medicine and acknowledge the Department of Health and the Minister for Health for their active engagement and willingness to finalize the agreement rapidly.”

Australians with CF join thousands of patients worldwide who are already receiving lumacaftor/ivacaftor. Beyond Australia, countries where lumacaftor/ivacaftor is reimbursed include Austria, Denmark, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S.

About CF
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people including countries within North America and Europe, as well as Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

About ORKAMBI ® (lumacaftor/ivacaftor) and the F508del mutation
In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

About Vertex
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.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Brazil. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Mr. Arbuckle’s statements in the second paragraph of this press release. While Vertex believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to commercializing ORKAMBI and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1-617-341-6108
or
Eric Rojas, +1-617-961-7205
or
Zach Barber, +1-617-341-6470
or
Media:
mediainfo@vrtx.com
or
North America:
Heather Nichols, + 1-617-341-6992
or
Europe & Australia:
Marie von Seyfried, + 44 7933 500887

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

MyHeritage er oppnevnt som hovedsponsor for the Eurovision Song Contest 201926.9.2018 12:09Pressemelding

MyHeritage, det ledende, globale slektshistorie og DNA-selskapet, er i dag blitt oppnevnt som hovedsponsor for Melodi Grand Prix 2019 av den Europeiske Kringkastingsunionen (EBU). MyHeritage har, som arrangementets hovedsponsor, fått omfattende global assosiasjon og arrangement, media og digitale rettigheter i forbindelse med den kommende sangkonkurransen som vil finne sted i Tel Aviv. Denne pressemeldingen inneholder multimedia. Se hele pressemeldingen her: https://www.businesswire.com/news/home/20180926005471/no/ MyHeritages tjenester gjør det lett for hvem som helst, hvor som helst, å begi seg ut på en fascinerende oppdagelsesferd for å finne sine røtter. Ved hjelp av MyHeritage DNA-testen, MyHeritages integrerte slektstre-bygging-platform (Family Tree Builder) og samlinger bestående av ni milliarder historiske dokumenter, hjelper MyHeritage millioner av mennesker til å oppdage sine familiers fortid, og til å forstå hvordan de selv passer inn i den globale menneskelige mosaikken. De

Calldorado Selected as a Finalist of the 2018 Engage Awards26.9.2018 12:02Pressemelding

Calldorado ApS are proud to announce their selection as a finalist in the Best Employee Centric Strategy category of the Engage Awards 2018 – the only customer and employee engagement industry awards in the UK. Calldorado, a completely self-funded company, has grown exponentially in an incredibly short period of time, and this is reflected in the finalist list where it is shortlisted alongside giant brands such as Virgin Media and Bacardi. “We are delighted to be a finalist in the Best Employee Centric Strategy category of these prestigious awards. Our ethos from the very beginning has been to mix experienced expertise with young talent fresh from education. To nurture and develop the movers and shakers of tomorrow, whose careers will grow alongside the company. We operate on values of mutual trust and transparent sharing and listening. Everyone’s opinion has equal value, which inspires collaboration, and encourages thinking that leads to changes and product innovations which drive our

Slice Labs Raises $20M in Extended Series A to Globalize its On-Demand Insurance Cloud Platform26.9.2018 11:00Pressemelding

Slice Labs, a leading on-demand insurance cloud platform provider, today announced it has raised an additional $20 million in Series A funding, led by The Co-operators with participation from XL Innovate, Horizons, Munich Re/HSB Ventures, SOMPO, Veronorte, the investment arm of Grupo Sura, and JetBlue Technology Ventures. This sizable, extended round will be used to execute on the higher than planned global demand for Slice Insurance Cloud Services (ICS). Slice launched in 2016 with a mission to change, improve and redesign insurance. The company’s Insurance Cloud Services (ICS) is transforming traditional insurers into digitally-driven carriers by providing them with a platform that enables them to quickly ideate, experiment, test and deploy new, on-demand insurance products. Top global insurers have launched on-demand insurance products using ICS, and Slice will use this new financing to continue expanding its offering on a global scale. “The ICS platform is rapidly growing as insure

ZephyrTel Welcomes Vasona to Its Portfolio of Software Solutions Serving the Global Telecommunications Sector26.9.2018 10:01Pressemelding

ZephyrTel, an ESW Capital software company dedicated to serving the global telecommunications industry, is pleased to announce it has acquired Vasona Networks, a leader in network performance and edge computing software for mobile operators, effective immediately. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180926005376/en/ Mike Shinya, CEO of ZephyrTel (Photo: Business Wire) ZephyrTel is building a world-class telco practice, to provide a multi-solution and services proposition to the global telco industry and leading enterprises. The company’s primary objective is to offer software and SaaS solutions across telco companies IT estates; from infrastructure right through to customer experience. This, coupled with high levels of industry domain expertise, will enable it to offer consultancy on both business strategy and technology execution. Having already acquired several telecom software companies, ZephyrTel is currently

Hallstar Names Scott Hinkle as New Chief Financial Officer and Head of Global Shared Services26.9.2018 10:00Pressemelding

Scott J. Hinkle has joined Hallstar, the global specialty chemistry company, as Chief Financial Officer. He will be based in Hallstar’s corporate headquarters in Chicago, Illinois, reporting directly to Chairman and CEO, John J. Paro. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180926005216/en/ Hallstar Names Scott Hinkle as New Chief Financial Officer and Head of Global Shared Services (Photo: Business Wire) Founded in 1986, Hallstar has become widely recognized for its innovative expertise in the areas of photostability science, renewable plasticizers, eco-based actives, phthalate replacements, natural olive chemistry and polymer modification. The company boasts research and development, manufacturing, and sales operations in China, Italy, France, and Brazil, in addition to the United States. Hallstar recently announced a structural split into Hallstar Beauty and Hallstar Industrial, effective October 1, 2018. These two

Global survey reveals that physicians need more information to assess the impact of treatment sequencing on patient survival in EGFR mutation-positive NSCLC26.9.2018 08:00Pressemelding

Results from a new global survey revealed that more than one-third (36%) of the 310 physicians surveyed do not think they have sufficient information required to make informed decisions on how to sequence treatments for patients with epidermal growth factor receptor mutation-positive (EGFR M+) non-small cell lung cancer (NSCLC). The survey results will be presented today as a late-breaking abstract (P3.01-108) at the 2018 World Conference on Lung Cancer (WCLC) in Toronto, Canada. The two most important treatment goals identified by those surveyed were increasing overall survival (OS) (77%), followed by quality of life (QoL) (69%), irrespective of the line of treatment. Findings show that more than half (55%) of physicians strongly preferred a treatment sequence that offers patients the maximum time on targeted treatments. Furthermore, physicians reported that there is a strong need for information on potential treatment resistance mutations before changing their current treatment pract