Business Wire

Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation

Jaa

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.

About Vertex

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.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach Barber, 617-341-6470
or
Media:
mediainfo@vrtx.com
or
Europe & Australia:
Rebecca Hunt, +44 7718 962 690
or
North America:
Megan Goulart, + 1-617-341-6992

Tietoja julkaisijasta

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 jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Emmaus Life Sciences Announces the New England Journal of Medicine has Published the Phase 3 Trial Results of Endari™ (L-Glutamine Oral Powder) in Sickle Cell Disease19.7.2018 00:45Tiedote

VIEW E-MEDIA KIT – Emmaus Life Sciences, Inc. (Emmaus) announced today that the New England Journal of Medicine (NEJM) has published the results of its 48-week phase 3 clinical trial of Endari™ (L-glutamine oral powder) which supported the FDA approval in July 2017 to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. The article reports results that showed significantly fewer sickle cell crises in those receiving Endari compared to placebo by 25 percent; p=0.005 (median 3 vs. median 4) and significantly fewer hospitalizations by 33 percent; p=0.005 (median 2 vs. median 3). Additional findings showed lower cumulative days in hospital of 41 percent; p=0.02 (median 6.5 days vs. median 11 days) and a lower incidence of acute chest syndrome (ACS) by more than 60 percent; p=0.003 (13 of 152 patients [8.6%] had at least 1 ACS compared with 18 of 78 in the placebo group [23.1%]). The most common adverse reactions, occurring in great

Tigo Releases State-of-the-art Wireless Technology – Mesh – as the New Solar Communication Architecture for the TS4 Platform18.7.2018 22:41Tiedote

Tigo®, pioneer of the smart modular Flex MLPE platform, today announced the release of its new Mesh communication architecture. This state-of-the-art wireless technology directly translates to customer benefits – including simplifying the solar design process and accelerating the commissioning steps. The complete Tigo solution uses a simple yet powerful data collection technology that covers the widest ranges of residential & commercial installations at the lowest cost. With Mesh and the recently announced Tigo Access Point (TAP), customers eliminate the need to address any roof obstruction or orientation constraint. To learn more about Mesh and TAP, register for Tigo’s free, online webinar “The Future of Solar is Wireless” on Wednesday July 25th, 2018 at 10am PDT. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180718005827/en/ Mesh is Tigo's proprietary software that allows each TS4 unit to act as a repeater, extending the

ViiV Healthcare Shares Data from Landmark 2-Drug Regimen Trials At AIDS 201818.7.2018 20:21Tiedote

ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. and Shionogi Limited as shareholders, will be presenting over 20 abstracts, including data from the landmark GEMINI 1 & 2 clinical trials, at the 22nd International AIDS Conference (AIDS 2018), 23-27 July 2018, in Amsterdam, The Netherlands. The data being presented this year has a strong focus on novel exploratory therapeutic options and innovative treatment strategies, as well as improving awareness and understanding of key issues that continue to affect the HIV community. John C Pottage, Jr, MD, Chief Scientific and Medical Officer, ViiV Healthcare, commented, “As we see advancements in the treatment options available to people living with HIV, our research efforts over the past few years have been focused on moving beyond viral load and addressing some of the unresolved issues that people living with HIV face, such as the potential long-term risks and toxicities that can be associated with li

Xilam: H1 2018 Revenue: +50%18.7.2018 19:00Tiedote

Regulatory News: The Xilam Group (Paris:XIL) posted consolidated H1 2018 revenue of EUR 13,866 thousand, an increase of 50%. This increase represents an excellent performance, as it follows on the heels of growth of 54% in H1 2017. Revenue for the first part of this financial year breaks down as follows. In thousands of euros 30.06.2018 (1) 30.06.2017 Change New productions 8,465 4,571 +85% Catalogue 5,385 4,652 +16% Other 16 16 +0% Revenue and subsidies 13,866 9,239 +50% (1) Unaudited data. Remarkable success for new releases +85% Excellent H1 growth was driven by the two main businesses, New Productions and Catalogue. In New Productions, H1 deliveries generated record revenue of EUR 8,465 thousand, an 85% increase compared with H1 2017, and confirmed Xilam’s position as a major European animation company. Catalogue revenue was up 16% compared with H1 2017, thanks again to traditional TV channels and digital platforms, in both France and abroad. A fast-paced delivery schedule Thanks t

Department for International Trade: Life Sciences at the Heart of UK Economy18.7.2018 15:38Tiedote

The Government will today (Wednesday 18 July) host the second in a series of roundtables with stakeholders from key life science businesses, demonstrating the importance of the sector to the UK economy. The Chancellor, International Trade Secretary, Business Secretary and Health and Social Care Secretary will be amongst those that meet with senior representatives from leading UK and international life sciences companies as the UK positions itself as the global home of health innovation, welcoming overseas investment and seeking to boost exports in the process. Discussions at the roundtable will focus on how the future of the Life Sciences sector will be supported by the delivery of our modern Industrial Strategy, ensuring that the UK is ‘open for business’ with a positive business environment, our ambitions for a comprehensive agreement with the EU on our future relationship and the development and implementation of our independent trade policy. To date, the government has engaged sign

Universal Laser Systems Expands Its Materials Database with 3M, Victrex and Dexmet Materials18.7.2018 15:05Tiedote

Universal Laser Systems (ULS) announces the addition of 3M™, Victrex® and Dexmet materials to its materials database, the most extensive repository of laser material processing parameters for materials in the range of 10 watts to 500 watts. The 3M, Victrex and Dexmet materials new to the ULS materials database were specifically added for laser processing with the ULTRA and XLS platforms, suited for high accuracy and precision laser cutting, laser ablation and laser surface modification. The materials include: 3M™ Extreme Sealing Tape 4412N 3M™ Thermally Conductive Silicone Interface Pad 5516 Victrex® APTIV® PEEK Film Dexmet PolyGrid® 8PTFE10-125ST Expanded PTFE Dexmet MicroGrid® AL 25 Expanded Aluminum Laser processing notes, describing the results of the laser-material interaction for these materials, are also available in the Materials Library on the ULS website to help potential customers explore the advantages of deploying laser technology within their manufacturing, research and d

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