Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene
Vertex Pharmaceuticals (Europe) Limited, today announced that the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. In addition, the European Medicines Agency’s Committee for Orphan Medicinal Products recently recommended the maintenance of orphan designation for tezacaftor/ivacaftor in combination with ivacaftor.
“The authorization of tezacaftor/ivacaftor in combination with ivacaftor is welcome news for European CF patients, their families and everyone involved in their treatment and care. This new medicine is especially important for patients with residual function mutations and those who do not tolerate ORKAMBI® (lumacaftor/ivacaftor),” said Harry Heijerman, Professor and Head of Department of Pulmonology at University Medical Centre Utrecht, The Netherlands.
The EU Marketing Authorization was based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017. Results showed treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function, as determined by absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1); with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo. The improvements in lung function showed a mean absolute change in ppFEV1 compared to placebo of 4.0 percentage points (P<0.0001) and 6.8 percentage points (P<0.0001) in EVOLVE and EXPAND respectively. The most common adverse reactions experienced by patients who received tezacaftor/ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.
“Today marks an important milestone for many CF patients in Europe, including those who so far have had no available option to treat the CFTR protein defect responsible for their disease,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “With today’s Marketing Authorization, we are rapidly moving towards treating 90 percent of CF patients.”
Tezacaftor/ivacaftor in combination with ivacaftor was approved by the U.S. Food and Drug Administration (FDA) in February 2018 and by Health Canada in June 2018. It is marketed as SYMDEKO™ in the U.S. and Canada.
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and 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 tezacaftor/ivacaftor and ivacaftor
Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.
About EVOLVE and EXPAND
Data from the two Phase 3 studies EVOLVE and EXPAND were published in the New England Journal of Medicine in November 2017, the studies enrolled approximately 750 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and a second mutation associated with residual CFTR activity. Across both studies, patients treated with tezacaftor/ivacaftor in combination with ivacaftor experienced statistically significant improvements in lung function, as determined by absolute change from baseline in ppFEV1. The treatment was generally well tolerated; the most common adverse reactions (≥10%) experienced by patients who received tezacaftor/ivacaftor with ivacaftor in the pooled, placebo-controlled Phase 3 studies were headache (14% versus 12% on placebo) and nasopharyngitis (12% versus 10% on placebo).
About orphan designation for medicines
Orphan designation is granted by the European Medicines Agency’s Committee for Orphan Medicinal Products to treatments which either address an existing unmet need or can provide significant benefit for people with life-threatening or chronically debilitating diseases, affecting a small number of patients.
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 Latin America. 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, as amended, including the quotes in the second and fourth paragraphs of this press release. While the company 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 SYMKEVI in Europe 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
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Tommy Hilfiger Calls on Social Entrepreneurs to Take on the Tommy Hilfiger Fashion Frontier Challenge21.3.2019 11:00:00 EET | Tiedote
Tommy Hilfiger, which is owned by PVH Corp. [NYSE: PVH], is pleased to announce that year two applications for the Tommy Hilfiger Fashion Frontier Challenge are now open. The global program aims to support entrepreneurial start-up and scale-up stage businesses developing solutions that promote inclusive and positive change in fashion. After a successful first year, the company is excited to continue to work on identifying opportunities that support the advancement of the fashion industry. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190321005068/en/ The prestigious jury panel and TOMMY HILFIGER Challenge winners at the Final Event. (Photo: Business Wire) “The first chapter of this global initiative was an inspiring journey that put a spotlight on incredible ideas that could change the lives of people through a more positive and inclusive fashion landscape,” said Tommy Hilfiger. “Through the Tommy Hilfiger Fashion Frontier
Elliott Statement on Proposal to Uniper21.3.2019 10:00:00 EET | Tiedote
Elliott Advisors (UK) Limited (“Elliott”) has written to the Management Board of Uniper SE (the “Company” or “Uniper”), to formally request the convocation of an Extraordinary General Meeting (“EGM”) with the express purpose of instructing management to prepare a lawful domination agreement with the Company’s largest shareholder Fortum Oyj (“Fortum”). Elliott believes the thus far ill-defined and ambiguous nature of the relationship between Uniper and Fortum has created an unsatisfactory and unsustainable dynamic, which is detrimental to Uniper. In Elliott’s view, the status quo – operational underperformance and pervasive uncertainty – if sustained, will risk further undermining the Company’s fundamental value. Elliott believes a timely shareholder vote to advance a domination agreement may resolve the prolonged uncertainty at Uniper and clarify the relationship between the Company and Fortum, such that value can be created for the Company and all stakeholders. Elliott believes that F
Key UK SEP Patent Win for TQ Delta20.3.2019 22:03:00 EET | Tiedote
TQ Delta LLC, a technology development and licensing firm, secured a judgment on March 18 by the High Court of Justice of England and Wales immediately enjoining ZyXEL Communications UK Ltd and its Danish parent company, ZyXEL Communications A/S, from further infringement of TQ Delta's European Patent (UK) 1 453 268. TQ Delta's patent relates to Digital Subscriber Line ("DSL") technology. In an earlier judgment, handed down on March 12, the Court found that the patent was valid and essential to the operation of ADSL2 and VDSL2. In that judgment, the Court held that the ZyXEL defendants have infringed and continue to infringe the patent. The Court also found that a second patent would have also been essential and infringed but was invalid. In its judgment earlier this week, the Court also found that the ZyXEL defendants were "holding out" against taking a licence to TQ Delta's portfolio of patents relating to DSL. It granted an immediate injunction and costs against the ZyXEL defendants
Lehman Brothers Treasury Announces the Successful Auction of a Portion of its Intercompany Claim against Lehman Brothers Holdings Inc.20.3.2019 21:15:00 EET | Tiedote
In connection with its previously announced partial wind-down, Lehman Brothers Treasury Co. B.V. in liquidation (“LBT”), through its U.S. counsel Kramer Levin Naftalis & Frankel LLP, announced that its placement agent, Seaport Loan Products LLC, successfully concluded an auction of a US$10,475,844,413 portion of LBT’s US$34,548,000,000 intercompany claim (the “Intercompany Claim”) against Lehman Brothers Holdings Inc. for a total gross purchase price of US$179,472,166. The sale is expected to close during the first week of April 2019. The net proceeds of the sale (after deducting various sale-related costs and expenses), together with certain available cash, will be used to fund a final distribution to creditors of LBT who do not receive substitute notes as a result of LBT’s prior solicitation process. For more information on the partial wind-down, including information about the Intercompany Claim sale, the final cash distribution, and anticipated timelines, an Information Notice has
Susan Ochs Joins PSB as Global Head, Financial Services20.3.2019 20:34:00 EET | Tiedote
PSB, a global research-based consultancy, announced today that Susan Ochs has joined the firm as Senior Vice President and Global Head, Financial Services, effective immediately. Ochs is based in New York and is responsible for the global development of the financial services practice and will contribute to other industry practices as well. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190320005671/en/ Susan Ochs, SVP, Global Head, Financial Services, PSB (Photo: Business Wire) “We are proud of the amazing brands we currently partner with across the financial sector,” said Mike Chuter, CEO, PSB, to whom Ochs reports. “With Susan’s combination of private and public finance expertise we’ll be able to deliver even more meaningful insights to our current and future partners. We are delighted to welcome Susan and are excited about the deep experience she brings.” Ochs has more than two decades of experience across the financial
Corsair Infrastructure Partners Announces Long Term Investments by HarbourVest Partners20.3.2019 17:35:00 EET | Tiedote
Corsair Infrastructure Partners (“CIP”), the global infrastructure investment business of Corsair Capital (“Corsair”), today announced that HarbourVest Partners (“HarbourVest”), a leading institutional investor active across multiple asset classes and geographies, has invested in three new CIP-managed funds established to deploy capital in the aviation, port, and road sectors. Earlier this year, CIP’s new funds closed on the acquisitions of shareholdings in three platform companies – the airport developer Vantage Airport Group, the Australian stevedore DP World Australia, and the Spanish toll-road operator Itínere Infraestructuras – from various selling shareholders including Gateway Infrastructure Investments, a legacy CIP-managed vehicle. The investments from HarbourVest come from a long-term pool of institutional capital dedicated to supporting infrastructure equity sponsors like CIP, and will play an important role in the execution of CIP’s growth strategies for all three platforms
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme