Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation in the European Union
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Union Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending extension of the Marketing Authorization for ORKAMBI® (lumacafator/ivacaftor) to children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. There are approximately 3,400 patients ages 6 through 11 eligible for this medicine in Europe.
“Cystic fibrosis is a systemic, multi-organ, progressive disease present from birth,” said David Gillen, M.D., Head of International Medical Affairs at Vertex. “This recommendation brings us closer to being able to help more people with CF who currently do not have a medicine to treat the underlying cause of their disease.”
Positive CHMP opinions are recommendations for approval to the European Commission, which has the authority to approve medicines for the European Union. The European Commission generally follows the recommendations of the CHMP and typically extends the Marketing Authorization within three months. In some countries, we have in place existing agreements that will make ORKAMBI available to this age group immediately following Marketing Authorization. In other countries, Vertex will begin the country-by-country reimbursement process following Marketing Authorization.
About Cystic Fibrosis
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 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. It is an oral pill taken every 12 hours - once in the morning and once in the evening.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI
ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should only be used in these patients. It is not known if ORKAMBI is safe and effective in children under 6 years of age.
Patients should not take ORKAMBI if they are taking certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; the seizure medicines phenobarbital, carbamazepine, or phenytoin; the sedatives/anti-anxiety medicines triazolam or midazolam; the immunosuppressant medicines everolimus, sirolimus, or tacrolimus; or St. John's wort.
Before taking ORKAMBI, patients should tell their doctor if they: have or have had liver problems; have kidney problems; have had an organ transplant; are using birth control (hormonal contraceptives, including oral, injectable, transdermal or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI. Patients should tell their doctor if they are pregnant or plan to become pregnant (it is unknown if ORKAMBI will harm the unborn baby) or if they are breastfeeding or planning to breastfeed (it is unknown if ORKAMBI passes into breast milk).
ORKAMBI may affect the way other medicines work and other medicines may affect how ORKAMBI works. Therefore, the dose of ORKAMBI or other medicines may need to be adjusted when taken together. Patients should especially tell their doctor if they take: antifungal medicines such as ketoconazole, itraconazole, posaconazole, or voriconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
When taking ORKAMBI, patients should tell their doctor if they stop ORKAMBI for more than 1 week as the doctor may need to change the dose of ORKAMBI or other medicines the patient is taking. It is unknown if ORKAMBI causes dizziness. Patients should not drive a car, use machinery, or do anything requiring alertness until the patient knows how ORKAMBI affects them.
ORKAMBI can cause serious side effects including:
High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI. The patient's doctor will do blood tests to check their liver before they start ORKAMBI, every three months during the first year of taking ORKAMBI, and annually thereafter. The patient should call the doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine; or confusion.
Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If a patient has poor lung function, their doctor may monitor them more closely when starting ORKAMBI.
An increase in blood pressure has been seen in some patients treated with ORKAMBI. The patient's doctor should monitor their blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI. For children and adolescents, the patient's doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of breath and/or chest tightness; upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose; gastrointestinal symptoms including nausea, diarrhea, or gas; rash; fatigue; flu or flu-like symptoms; increase in muscle enzyme levels; and irregular, missed, or abnormal menstrual periods and heavier bleeding.
Please click here to see the full U.S. Prescribing Information for ORKAMBI.
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 and VX-659 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, including, without limitation, Dr. Gillen’s statement in the second paragraph of this press release and statements regarding the European Commission generally following the recommendations of the CHMP and typically issuing marketing approval within three months. While Vertex 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, that Vertex could experience unforeseen delays in obtaining marketing approval from the European Commission 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 the company'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
Megan Goulart, + 1-617-341-6992
Europe & Australia:
Rebecca Hunt, +44 7718 962 690
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
FCF: Greenpeace Allegations Unfair and Deceptive26.5.2018 18:02 | Pressemelding
FCF Fishery, Co. President Max Chou today released the following statement regarding recent misleading Greenpeace human rights abuse allegations made against the company: “We realize that as one of the world’s largest marine products integrated supply chain service providers with more than 30 subsidiaries, fishing bases, and shipping agents around the world, we are a prime target for organizations seeking to garner publicity and those not fully aware of the latest development and improvements that are currently taking place in the fishery industry. However, in their efforts to curb egregious human rights abuses, we believe it is unfair and deceptive to lump our company in with those who condone cruelty and neglect of their laborers. “Although we recognize the meaningful work of Greenpeace in exposing and eliminating human trafficking and sustainability abuses, we are equally disappointed that they are implicating FCF in old incidents and cases that have since been in all instances addr
NBC Universal, Havas Group and Endemol Shine Discuss a Future with AI, Blockchain and VR at IBC201825.5.2018 18:20 | Pressemelding
As is fitting for the home of technical invention and innovation, IBC2018 will present a broad range of ambitious and timely elements to engage, inform and inspire content creators and owners from around the world that will visit the RAI, Amsterdam from 13-18 September 2018. The must-attend event includes fresh inspiring conference themes that will focus on innovative new platforms, audience engagement and interaction/immersion, as well as a wide-ranging exhibition that will encompass all the latest technologies from AI and VR to advanced cloud-based workflows. Registration for IBC2018 is now open at: https://ibc.itnint.com/IBC18/Online/RegLogin.aspx “The media landscape is evolving at an unprecedented rate, with new platforms, technologies and even new realities at our disposal that offer greater opportunities for content innovation and audience engagement,” said Michael Crimp, CEO, IBC. “IBC is preparing an outstanding programme of conference sessions, forums and exhibition features
Pavilion of France at the 16th International Architecture Exhibition – La Biennale di Venezia25.5.2018 09:26 | Pressemelding
The concept for Infinite Places , developed by the Encore Heureux team, led by the architects Nicola Delon, Julien Choppin, and Sébastien Eymard, presents places produced by new and inventive processes that generate architectural processes of value. This exhibition project, which considers the territory as a whole, highlights initiatives on the part of civil society and communities that embody a certain free spirit of experimentation and the possibilities opened up by architecture. These projects are expressions of programmatic freedom and generosity, and they live up to the theme of Freespace adopted by the two commissioners of the 16th International Architecture Exhibition – La Biennale di Venezia, the Irish architects Yvonne Farrell and Shelly McNamara. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180525005191/en/ French Pavilion Venice Architectural Biennale, copyright S. Scher Statement of the curators « Through the I
SIA Acquires Card Processing Businesses in Central and Southeastern Europe from First Data25.5.2018 08:30 | Pressemelding
SIA, a European high-tech leader in payment infrastructure and services, and First Data Corporation (NYSE: FDC), a global leader in commerce-enabling technology, have signed an agreement for SIA to acquire First Data’s card processing businesses in parts of Central and Southeastern Europe for €375 million. In 2017, these businesses generated a combined revenue of approximately €100 million for First Data. This acquisition by SIA provides card processing, card production, call center and back-office services, including 13.3 million payment cards, 1.4 billion transactions, in addition to the management of POS terminals and ATMs. These businesses are primarily located in 7 countries: Greece, Croatia, Czech Republic, Hungary, Romania, Serbia and Slovakia. As a result of the transaction, SIA will become a leading player in processing and services in the region. The agreement includes the transfer of about 1,400 First Data employees into SIA. “This acquisition is in line with our strategy to
Westinghouse Accident Tolerant Fuel Development Moves Forward with Cooperation Agreement with ENUSA25.5.2018 06:00 | Pressemelding
Westinghouse Electric Company today announced that it will collaborate in the development of its EnCore® Fuel, the revolutionary accident-tolerant fuel (ATF) design, with ENUSA Industrias Avanzadas (ENUSA) through a Frame Cooperation Agreement (FCA). “This agreement serves to strengthen the technical and commercial relations between ENUSA and Westinghouse as we work to develop leading nuclear fuel technology,” said Torbjörn Norén, European Fuel Group and EMEA Fuel Delivery Director at Westinghouse. “Westinghouse’s work with ENUSA in the Spanish and European Fuel Group markets will help to facilitate agreements with customers to launch EnCore Fuel demonstration programs in their plants.” Under the terms of the agreement, the newly signed FCA establishes the framework that will regulate the different Joint Development Programs (JDPs) to be launched between both companies. The first JDP will evaluate the application of the segmented rod concept and develop models of ATF / EnCore fuel beha
BRIC HOLDING Offers Aimedis – ICO: Germany’s Leading eHealth Platform Goes Public25.5.2018 06:00 | Pressemelding
Since Wednesday, May 16, investors finally have a chance to claim their share of a unique success story, the international market roll-out of Aimedis, by purchasing AIM tokens: AIM tokens are now available with a 20% presale bonus PLUS a 5% early bird-premium only at the Czech investment house BRIC INVEST https://www.bric-holding.com/home_en.html! Aimedis aims to shape the future of healthcare by providing an advanced and fully operational platform, secured by blockchain and supported by AI, that enables patients to take control, secure the exchange of their medical data, access the best healthcare providers, get advice, prescriptions, personal health upgrades and to become part of the health revolution. And we are not talking about the future: The revolutionary Aimedis platform is already deployed as a live working system and is already in use in major hospital groups in Germany! By issuing 300 million AIM tokens at a nominal value of 0,12 USD each, the Aimedis consortium will raise c