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
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.
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.
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.
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.
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Eric Rojas, +1-617-961-7205
Zach Barber, +1-617-341-6470
Heather Nichols, + 1-617-341-6992
Europe & Australia:
Marie von Seyfried, + 44 7933 500887
Om Business Wire
(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
OCP Announces Date of Third Quarter 2018 Earnings20.11.2018 17:00 | Pressemelding
OCP S.A. (“OCP” or the “Company”), a global leader in the fertilizer industry, will release its third quarter 2018 results on Thursday, November 29, 2018. The results will be available to holders of the Company’s bonds, qualified institutional buyers, securities analysts and market makers on the OCP Intralinks portal from 8:30 a.m. EST, 2:30 p.m. Morocco time (GMT+1), and 1:30 p.m. London time (GMT). OCP senior management will host a conference call to discuss third quarter 2018 results at 9:30 a.m. EST, 3:30 p.m. Morocco time (GMT+1), and 2:30 p.m. London time (GMT) on Thursday, November 29, 2018 for holders of the Company’s bonds, qualified institutional buyers, securities analysts and market makers. Eligible parties that have not already registered for access to the Intralinks portal may do so by contacting the Investor Relations Department by emailing firstname.lastname@example.org About OCP OCP is a global leader in the fertilizer industry, backed by almost a century’s production history. O
Arch Insurance Announces Strategic Leadership Changes20.11.2018 15:50 | Pressemelding
Arch Insurance (“Arch”) today announced Hugh Sturgess will assume the role of Chief Executive Officer, Arch Insurance International, effective Jan. 1, 2019, subject to regulatory approvals. Arch Insurance International includes Arch’s insurance operations in Europe, Bermuda and Australia. Mr. Sturgess has been with Arch Insurance since 2005 and is currently President and CEO of Arch Insurance Canada Ltd., where he has strategic and operational responsibility for a multi-line underwriting portfolio. Before joining Arch, Mr. Sturgess held various roles in the financial services industry, including tenures with the Royal Bank of Canada and Chubb Insurance Company of Canada. “Arch Insurance International underwrites a diverse portfolio of specialty insurance solutions across a number of geographies, with a key focus on providing value to our distribution partners and customers,” Mr. Sturgess said. “I look forward to the opportunity to work with our team in continuing to expand the value pr
Takeda Receives Clearance from the European Commission for the Proposed Acquisition of Shire plc20.11.2018 14:33 | Pressemelding
NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN ANY JURISDICTION WHERE TO DO SO WOULD CONSTITUTE A VIOLATION OF THE RELEVANT LAWS OF SUCH JURISDICTION Takeda Pharmaceutical Company Limited (“Takeda”) announces that it has received clearance from the European Commission (the “EC”) for the proposed acquisition of Shire plc (“Shire”) announced on May 8, 2018 (the “Acquisition”). The EC’s approval is conditional on Takeda and Shire fulfilling commitments given to the EC in connection with the clearance. Specifically, in relation to the future potential overlap in the area of inflammatory bowel diseases between Takeda’s marketed product Entyvio (vedolizumab) and Shire’s pipeline compound SHP647, Takeda and Shire have committed to divest the pipeline compound SHP647 and certain associated rights. The divestment of SHP647 and certain associated rights is not a condition to the completion of the Acquisition. SHP647 is an exciting pipeline compound
ResMed Elects Harjit Gill to Its Board of Directors20.11.2018 14:05 | Pressemelding
ResMed (NYSE: RMD, ASX: RMD), a leader in cloud-connected medical devices and out-of-hospital software-as-a-service business solutions, today announced it has elected experienced consumer and healthcare executive Harjit Gill to its Board of Directors. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181120005179/en/ Harjit Gill, BA (Photo: Business Wire) Ms. Gill was elected by shareholders at its annual shareholder meeting last week in Sydney, Australia. Also, announced at that meeting, Gary W. Pace has retired from ResMed’s board after serving on it since July 1994. Ms. Gill’s appointment and Dr. Pace’s retirement both took effect Thursday, November 15. Ms. Gill has more than 25 years’ experience in health and consumer technology products as an executive, investor and regional business leader spanning Asia, Europe and the Middle East. From 1990 to 2015, she worked for Royal Philips as executive vice president and CEO for Phi
PTC Adds Artificial Intelligence and Generative Design Capabilities to Enhance and Expand its CAD Portfolio with Acquisition of Frustum20.11.2018 12:12 | Pressemelding
PTC (NASDAQ: PTC) today announced that it has acquired Frustum Inc., an advanced generative design software company, for approximately $70 million. Frustum’s technology for generative design—an approach that leverages artificial intelligence (AI) to generate design options—is a transformative addition to the PTC Creo portfolio. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181120005326/en/ PTC Adds Artificial Intelligence and Generative Design Capabilities to Enhance and Expand its CAD Portfolio with Acquisition of Frustum (Graphic: Business Wire) Based in Boulder, Colorado, Frustum offers patented desktop and cloud-based engineering software that enables designers and engineers to go beyond the limits of their personal experience by leveraging powerful AI capabilities that guide the discovery of high-performance, next-generation product designs. “PTC is pushing the boundaries of innovation with this acquisition,” said Jim
United First Partners Launches UFP Fintech20.11.2018 12:00 | Pressemelding
United First Partners (“UFP”), a leading independent Research, Brokerage, Advisory & Investment group is pleased to announce the launch of its latest venture: UFP Fintech. UFP Fintech invests in early stage Fintech, Proptech and Legaltech startups. Under the leadership of the entity’s CIO Christophe Petetin, a veteran investor in the Fintech space with an unparalleled investment track record in Fintech start-ups, UFP Fintech aims to become a global leader in Early Stage Fintech Investments. In recent years, Christophe and the UFP Fintech team have seeded a major unicorn and several other market leading entities. Christophe is a premier Fintech thought leader, who also acts in an advisory capacity to governmental and regulatory bodies, such as the FCA and ISO 20022 to create the next generation of standards for Fintech, payments, data, securities & digital currencies. Christophe commented “The financial industry is getting rebuilt from the ground up creating unprecedented opportunities.