Vertex Receives European CHMP Positive Opinion for SYMKEVI® (tezacaftor/ivacaftor) for People with Cystic Fibrosis Aged 12 and Older with Certain Mutations in the CFTR Gene
Vertex Pharmaceuticals (Europe) Limited, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion 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.
If granted Marketing Authorization by the European Commission (EC), tezacaftor/ivacaftor will be used in combination with ivacaftor and will be the first medicine to treat the CFTR protein defect in CF patients who have one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity. It also provides a new treatment option for a significant number of people living with CF who have two copies of the F508del mutation.
“Our goal at Vertex is to find a cure for all people living with CF and we are moving rapidly towards treating up to 90 percent of patients,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Today’s announcement is a pivotal accomplishment along that journey. If approved, tezacaftor/ivacaftor in combination with ivacaftor represents an important option for people with two copies of the F508del mutation and the first medicine in the EU for patients with one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity.”
The regulatory submission was supported by 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 ppFEV1, and with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo. 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.
"Tezacaftor/ivacaftor combination therapy for CF is another important achievement in the development of disease modulating therapies. This combination improves important clinical outcomes and may benefit those who cannot use ORKAMBI® (lumacaftor/ivacaftor),” said Professor Stuart Elborn, Clinical Professor of Respiratory Medicine, and Centre Director for Specialist Adult Cystic Fibrosis at the Royal Brompton Hospital, London.
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.
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 second mutation that is responsive to tezacaftor/ivacaftor. 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 percent predicted forced expiratory volume in one second (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).
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 and Australia. 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. For additional information and the latest updates from the company, please visit www.vrtx.com.
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:
Rob Clark, + 44 7736 473227
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
P&G Launches New Film Highlighting Its Internal Journey for LGBT+ Inclusion18.6.2019 18:00:00 CEST | Pressemelding
The Procter & Gamble Company (NYSE:PG) in partnership with Great Big Story, today released Out of the Shadows, a new film chronicling P&G’s journey of Lesbian, Gay, Bisexual and Transgender inclusion. The film highlights the employees who challenged the Company and overcame adversity during the tumultuous 1990s and early 2000s. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190618005920/en/ P&G, in partnership with Great Big Story, today released Out of the Shadows, a new film chronicling P&G’s journey of Lesbian, Gay, Bisexual and Transgender inclusion. The film highlights the employees who challenged the company and overcame adversity during the tumultuous 1990s and early 2000s. (Photo: Business Wire) In 1992, P&G became one of the first Fortune 500 companies to add sexual orientation into its diversity statement. The struggle for workplace equality for the LGBT+ community, however, was just beginning. Ravaged by the HIV/A
Rockwell Automation Opens New Electric Vehicle Innovation Center in Silicon Valley18.6.2019 14:00:00 CEST | Pressemelding
Rockwell Automation, a leading solutions and services provider to the automotive industry, today opened a new 8,000 square-foot Electric Vehicle (EV) Innovation Center at 111 North Market Street in San Jose, California, within its Information Solutions development facility. The center will provide live manufacturing demonstrations, hands-on trials utilizing new technology and events showcasing collaboration with industry experts and Rockwell Automation partners. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190618005201/en/ Utilizing augmented and virtual reality modeling, the EV Innovation Center provides automotive start-ups and established manufacturers an environment to learn new technologies and standards, enabling them to deliver electric vehicles to market faster, with less risk and at lower cost. The combination of Rockwell Automation technology with partner technology is what makes the center unique. Specifically,
Andersen Name Grows in Poland18.6.2019 13:30:00 CEST | Pressemelding
Just a few months after joining Andersen Global as a collaborating firm, KSP Legal and Tax Advice (KSP T.Srokosz i Wspólnicy Sp.k.), has adopted the name Andersen Tax & Legal and joined the Andersen Tax & Legal team in Poland. The Andersen name now has broad coverage in Poland with Andersen Tax & Legal offices in both Warsaw and Katowice. Marcin Matyka will be Managing Partner of Andersen Tax & Legal in Warsaw, while the team in Katowice will continue to be led by Office Managing Partner Magdalena Patryas. “We are excited to adopt the Andersen name and be a part of a global organization where we can provide best-in-class services in a seamless manner across the globe,” said Magdalena. “Very early in our relationship it was obvious that our group was very much aligned with Andersen values and the mission of providing the best in tax and legal services, what we have been doing in Katowice for the last 16 years.” The Katowice-based group has over 30 legal and tax advisors who provide lega
Ascend Completes Investment in Reliability, Capacity of NTA Chelate Production18.6.2019 13:00:00 CEST | Pressemelding
Ascend Performance Materials, a global provider of premium chemicals, fibers and plastics, announced today the completion of a multimillion-dollar reliability and capacity investment in nitrilotriacetic acid production at its Chocolate Bayou facility in Alvin, Texas. The investment increased production of the company’s biodegradable chelate, FlexaTrac™-NTA, by over 10 percent. “Demand for our FlexaTrac-NTA has increased by nearly 30 percent over the past two years,” said Jonathan Craig, vice president of specialty chemicals at Ascend. “Our continued investment in capacity and production is about providing our customers with a biodegradable, cost-effective solution that meets their needs.” FlexaTrac-NTA is a highly effective chelating agent used primarily in industrial cleaning and oil and gas applications to bind to and isolate heavy metal ions. NTA has a higher binding capacity than other widely used chelates currently available in the market. About Ascend Performance Materials Ascend
DXC Technology Launches Innovation Centre in London18.6.2019 13:00:00 CEST | Pressemelding
DXC Technology (NYSE: DXC), the world’s leading independent, end-to-end IT services company, today announced the opening of the DXC Innovation Centre in London for the company and its clients to develop and deliver high-impact digital solutions. DXC’s London Innovation Centre is a collaborative environment that draws on the company’s global experience in digital transformation, leverages its rich industry knowledge and independent partner network, and creates an environment for the incubation of ideas, learning and development. The London Innovation Centre adds to DXC’s digital network in the UK, which includes a Digital Transformation Centre in Newcastle that is already helping customers to transform and accelerate the creation of business value. As part of this network, DXC digital technologists and enterprise solution experts will work with customers and partners to modernize and integrate mainstream IT by deploying digital solutions at scale. “Our Innovation Centre brings together
Small Giant Games Announces Epic Expansion to Hit Franchise Empires & Puzzles18.6.2019 13:00:00 CEST | Pressemelding
Small Giant Games (“Small Giant”), a subsidiary of Zynga Inc. (Nasdaq: ZNGA), a global leader in interactive entertainment, is announcing an epic expansion to its popular franchise, Empires & Puzzles. The expansion, backed by player demand, features significant base updates including an increase of the stronghold’s max level from 20 to 21 as well as advanced versions of mines, farms, storage units and houses. A brand new building, the Hunter’s Lodge, is also being introduced, allowing players to craft new battle items. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190618005014/en/ Empires & Puzzles from Small Giant Games (Graphic: Business Wire) “Since the Small Giant team joined the Zynga family last year, we’ve been blown away by their continued ingenuity in innovating within Empires & Puzzles, a cornerstone of our portfolio of forever franchises,” said Bernard Kim, President of Publishing at Zynga. “Comprised of passiona