Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation, to include children ages 6 through 11. In Europe, there are approximately 3,400 children ages 6 through 11 with two copies of this mutation. Existing reimbursement agreements in countries such as Ireland will enable rapid access to ORKAMBI for these children. In other countries across the European Union, Vertex will now begin the country-by-country reimbursement process.
“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to ORKAMBI,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”
The European Commission’s decision is based on data from two Phase 3 studies of ORKAMBI in children with CF ages 6 through 11 who have two copies of the F508del mutation. In 2017, The Lancet Respiratory published 24-week data from one of these studies, which demonstrated statistically significant improvements in lung function (as assessed by the absolute change in lung clearance index, or LCI2.5, and predicted forced expiratory volume in one second, or ppFEV1) among children treated with ORKAMBI compared to placebo. Improvements in body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score were also observed, although they were not statistically significant. Safety data were similar to those observed in an earlier Phase 3 open-label safety study in children ages 6 through 11. The most common adverse events that occurred more frequently among those receiving ORKAMBI compared to placebo were infective pulmonary exacerbation, productive cough, nasal congestion, oropharyngeal pain, abdominal pain upper, headache, upper respiratory tract infection and sputum increased.
“A principal goal of treating CF is slowing the progressive lung damage caused by this life-shortening genetic disease while improving health in the short term,” said Professor Marcus Mall, M.D., Director of the Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis Center at the Heidelberg University Hospital, Germany. “Studies of ORKAMBI in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.”
About Cystic Fibrosis (CF)
CF is a rare, life-shortening genetic disease affecting approximately 75,000 people across 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 protein 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 buildup 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)
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. ORKAMBI 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 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.
Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), tezacaftor, VX-440, VX-152, VX-659 and VX-445 were discovered by Vertex as part of this collaboration.
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 and statements regarding the timing of access to ORKAMBI for patients 6 through 11 and the country-by-country reimbursement approval process. 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 ORKAMBI for patients 6 through 11 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 and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Pharmaceuticals Incorporated
Michael Partridge, 617-341-6108
Eric Rojas, 617-961-7205
Zach Barber, 617-341-6470
Europe & Australia:
Rebecca Hunt, +44 7718 962 690
Megan Goulart, + 1-617-341-6992
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
Warm welcome for ABB Formula E in Davos19.1.2019 12:00 | Tiedote
Just a few days before the start of the World Economic Forum 2019, Davos is celebrating a world premiere: The latest ABB FIA Formula E racing car - dubbed Gen2 – was transported on a train from the iconic Swiss Rhaetian Railway to Europe’s highest city, where it will be an exciting eyecatcher during the coming week at the Davos Platz railway station. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190119005003/en/ ABB FIA Formula E racing car in Davos (Photo: Business Wire) From this Saturday until next Sunday, January 27, the Gen2 will be on display in a special glass case. Interested passersby can learn about the ABB FIA Formula E racing series as well as about the activities of ABB in e-mobility via an information system. With over 8,500 fast charging stations installed in 70 countries around the world, ABB is the global market leader for charging infrastructures. To watch the video, click here ABB (ABBN: SIX Swiss Ex) is
PMI’s Mission Winnow Goes Full Throttle with Ducati Corse for 2019 MotoGP™18.1.2019 20:00 | Tiedote
Philip Morris International Inc. (PMI) (NYSE: PM) is pleased to announce that its Mission Winnow initiative is expanding in 2019 to include the Ducati Corse racing team. Mission Winnow is a PMI-led campaign to raise global awareness of our passion and determination to constantly improve and evolve, as well as highlight the power of science, technology and innovation to build a better future. PMI has partnered with Ducati Corse since 2002 and has extended the relationship for another three years until the end of 2021. As of the start of this year’s MotoGP season, the team will be officially known as Mission Winnow Ducati. Mission Winnow was first launched in October 2018 with Scuderia Ferrari Mission Winnow, which – like Ducati Corse – has a passion for innovation and a relentless drive to improve in the team’s pursuit of victory. Ducati Corse is one of the most inspiring and resilient teams in MotoGP, with a 70-year history in racing. Ducati fans form a passionate community who appreci
IFF’s Frutarom Division Completes Acquisition of 60% of Thailand-based Mighty18.1.2019 15:50 | Tiedote
Regulatory News: International Flavors & Fragrances Inc. (NYSE:IFF) (Euronext Paris:IFF) (TASE:IFF), announced that its Frutarom Division has completed the acquisition of 60% of the share capital of The Mighty CO. LTD. (“Mighty”), a leading savory solutions provider in Thailand, thus continuing its growth strategy in Southeast Asia. Amos Anatot, President of IFF's Frutarom Division, said, “The completion of this deal with Mighty underscores that the Frutarom division will continue on its growth strategy and pursue attractive companies that create new opportunities or build on current capabilities.” Mr. Anatot continued, “And in this case, we are helping to grow our capabilities in savory solutions – already an area of strength for legacy Frutarom, now IFF." Mighty, founded in 1989, develops, produces and markets reaction flavors, with particular expertise in savory solutions. The company’s portfolio includes flavors, seasoning blends, marinades, and specialty functional raw materials f
LTI Q3 FY19: Constant currency revenue growth up 6.1% QoQ and 20.6% YoY; Net Profit jumps 32.8% YoY18.1.2019 15:02 | Tiedote
Larsen & Toubro Infotech (BSE code: 540005, NSE: LTI), a global technology consulting and digital solutions company, announced its Q3 FY19 results today. In US Dollars: Revenue at USD 346.9 million; growth of 5.6% QoQ and 18.2% YoY Constant Currency Revenue growth of 6.1% QoQ and 20.6% YoY In Indian Rupees: Revenue at Rs 24,729 million; growth of 6.1% QoQ and 31.3% YoY Net Income at Rs 3,755 million; growth of (6.2%) QoQ and 32.8% YoY “We are pleased to deliver another strong quarter with 5.6% QoQ growth in USD revenues. Our broad-based revenue growth, superior margin delivery and steady cash generation in Q3 is a testimony of our focused execution and client centricity. We are also thrilled to welcome Ruletronics to LTI family. Ruletronics enables businesses to transform and evolve digitally by providing innovative BPM and CRM solutions leveraging Pega Platform.” - Sanjay Jalona, Chief Executive Officer & Managing Director, LTI Recent Deal Wins Nets, the leading payments company in th
Schlumberger Announces Full-Year and Fourth-Quarter 2018 Results18.1.2019 15:00 | Tiedote
Schlumberger Limited (NYSE: SLB) today reported results for full-year 2018 and the fourth quarter of 2018. (Stated in millions, except per share amounts) Full-Year Results Twelve Months Ended Change Dec. 31, 2018 Dec. 31, 2017 Year-on-year Revenue $32,815 $30,440 8% Pretax operating income $4,187 $3,921 7% Pretax operating margin 12.8% 12.9% -12 bps Net income (loss) - GAAP basis $2,138 $(1,505) n/m Net income, excluding charges & credits* $2,261 $2,085 8% Diluted EPS (loss per share) - GAAP basis $1.53 $(1.08) n/m Diluted EPS, excluding charges and credits* $1.62 $1.50 8% Full-Year Consolidated Revenue by Area North America $11,984 $9,487 26% Latin America 3,745 3,976 -6% Europe/CIS/Africa 7,158 7,072 1% Middle East & Asia 9,543 9,394 2% Other 385 511 n/m $32,815 $30,440 8% North America revenue $11,984 $9,487 26% International revenue $20,446 $20,442 - North America revenue, excluding Cameron $9,668 $7,518 29% International revenue, excluding Cameron $17,675 $17,423 1% *These are non
FLIR Systems Awarded $89 Million Contract from French Armed Forces to Deliver Black Hornet Personal Reconnaissance System18.1.2019 14:00 | Tiedote
FLIR Systems, Inc. (NASDAQ: FLIR) announced today it has been awarded a contract from the French Defense Procurement Agency (DGA) in support of the French Operational Pocket Drone (DrOP) program. The contract has a ceiling value of $89 million to provide the FLIR Black Hornet® 3 nano-unmanned aerial vehicle (UAV) and Personal Reconnaissance System (PRS) to support French Armed Forces operations. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190118005085/en/ The French Armed Forces awarded FLIR Systems a contract to deliver the Black Hornet Personal Reconnaissance System for military operations. (Photo: Business Wire) The Black Hornet PRS is the world’s smallest combat-proven nano-Unmanned Aerial System (UAS) and is currently deployed in more than 30 countries. The Black Hornet enables the warfighter to maintain situational awareness, threat detection, and surveillance no matter where the mission takes them. Equipped with el
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