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
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
Manchester United and True Religion Launch Denim Range19.10.2018 20:37 | Tiedote
Manchester United (NYSE:MANU) and luxury denim brand True Religion have collaborated to launch a range of premium club branded denim wear. A first for the club, the new global partnership with the iconic American denim brand will see a range of men’s & women’s co-branded styles go on sale beginning 26th October in the club’s Megastore as well as online via United Direct and truereligion.com & eu.truereligion.com. The exclusive collection features jeans, shirts and jackets, including a highly desirable limited edition denim jacket embroidered with the club’s crest. Fans will have the opportunity to win a selection of clothing from the new range by visiting www.manutd.com/truereligion. Manchester United’s Group Managing Director, Richard Arnold, comments: “True Religion is a well-known, established name in fashion, creating unique designs without compromising on quality. The range we have collaborated on includes the same attention to detail and craftsmanship that has made True Religion
Arch Insurance Announces Strategic Leadership Changes19.10.2018 16:10 | Tiedote
Arch Insurance today announced that Matt Shulman will assume the newly created role of CEO, Arch Insurance North America, effective January 1, 2019. In this role, he will lead Arch Insurance’s operations in the United States and Canada. He will report to Nicolas Papadopoulo, Chairman and CEO of Arch Worldwide Insurance Group. Mr. Shulman, who has more than 20 years of experience in the insurance industry, has been with Arch Insurance since 2009 and has served as the President and CEO of Arch Insurance Europe since 2016. “Matt brings significant U.S. and international experience to this role. Under his leadership, together with our senior team, Arch Insurance will continue to enhance our value proposition to our customers through a robust, diversified product portfolio, creative solutions and excellent service,” Mr. Papadopoulo said. Arch Insurance has also created a new organizational structure with three Chief Underwriting Officers (CUO) dedicated to specific lines of business. These
Takeda to Present Results from Phase 3 ALTA-1L Trial Highlighting Intracranial Efficacy of ALUNBRIG® (brigatinib) Versus Crizotinib in First-Line Advanced ALK+ Non-Small Cell Lung Cancer19.10.2018 15:00 | Tiedote
Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that intracranial efficacy data from the Phase 3 ALTA-1L (ALK in Lung Cancer Trial of BrigAtinib in 1 st Line) trial showed improved intracranial progression-free survival (PFS) and intracranial objective response rate (ORR) with ALUNBRIG (brigatinib) compared to crizotinib among anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) patients. Data for these secondary endpoints will be presented in a poster discussion at the European Society for Medical Oncology (ESMO) 2018 Congress on Friday, October 19 at 2:00 p.m. CET in Munich, Germany. These results further support ALUNBRIG as a potential treatment for adults with ALK+ locally advanced or metastatic NSCLC who had not received a prior ALK inhibitor. ALUNBRIG is currently not approved as first-line therapy for advanced ALK+ NSCLC. “ALK+ NSCLC often spreads to the brain, so having options that can clearly demonstrate efficacy both in the brain an
Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) to Treat Patients With Cystic Fibrosis Aged 12 to <24 months With Certain Mutations in the CFTR Gene19.10.2018 14:54 | Tiedote
Vertex Pharmaceuticals (Europe) Limited today announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KALYDECO® (ivacaftor) to include the treatment of people with cystic fibrosis (CF) aged 12 to <24 months who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. If the European Commission issues a favorable adoption of the EMA CHMP opinion for the extension of indication, ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF in patients aged 12 to <24 months, who have specific mutations in the CFTR gene. “Cystic fibrosis is a chronic, progressive disease that is present at birth, with symptoms often occurring in infancy, so early treatment is crucial to deliver the best possible outcomes for patients,” said Reshma Kewalr
Tradeshift Announces Q3 2018 Results19.10.2018 14:00 | Tiedote
Tradeshift, the leader in supply chain payments and marketplaces, today announced results from the third quarter of 2018, marking the tenth quarter in a row of successive growth and beating targets. Tradeshift’s third quarter growth stats announced today include: YoY revenue grew 400 percent (trailing 12 months) YoY new bookings grew 284 percent YoY gross merchandise volume (GMV) grew 262 percent New total contract value grew by $47M in Q3 Tradeshift’s customer roster continued strong growth this quarter adding 27 new customers, including Hertz, Shiseido, ECU, and Fortune 500 leaders in retail apparel, agriculture, engineering and construction, hospitality, travel and food delivery. Tradeshift also expanded its app ecosystem by adding a key partnership with Coface, a global credit insurer. Coface has announced a new app solution on the Tradeshift platform offering risk and business information services to help businesses make decisions by ensuring greater financial transparency between
Schlumberger Announces Third-Quarter 2018 Results19.10.2018 14:00 | Tiedote
Schlumberger Limited (NYSE: SLB) today reported results for the third quarter of 2018. (Stated in millions, except per share amounts) Three Months Ended Change Sept. 30, 2018 Jun. 30, 2018 Sept. 30, 2017 Sequential Year-on-year Revenue $8,504 $8,303 $7,905 2% 8% Pretax operating income $1,152 $1,094 $1,059 5% 9% Pretax operating margin 13.5% 13.2% 13.4% 36 bps 15 bps Net income - GAAP basis $644 $430 $545 50% 18% Net income, excluding charges & credits* $644 $594 $581 8% 11% Diluted EPS - GAAP basis $0.46 $0.31 $0.39 48% 18% Diluted EPS, excluding charges & credits* $0.46 $0.43 $0.42 7% 10% North America revenue $3,189 $3,139 $2,602 2% 23% International revenue $5,215 $5,065 $5,147 3% 1% North America revenue, excluding Cameron $2,572 $2,546 $2,086 1% 23% International revenue, excluding Cameron $4,559 $4,387 $4,430 4% 3% *These are non-GAAP financial measures. See section below entitled "Charges & Credits" for details. Schlumberger Chairman and CEO Paal Kibsgaard commented, “Our third
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