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
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 tältä julkaisijalta, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
The 6th Annual World Patient Safety, Science & Technology Summit Kicked off with an Important Announcement about the Progress Being Made to Save Lives in Hospital Settings24.2.2018 13:00 | Tiedote
The 6th Annual World Patient Safety, Science & Technology Summit kicked off with an important announcement about the progress being made to save lives in hospital settings. Nearly 4,600 hospitals in 44 countries have committed to adopting patient safety processes that are proven to eliminate preventable deaths in hospitals. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180224005027/en/ England’s Health and Social Care Secretary, Rt. Hon. Jeremy Hunt, launches groundbreaking new measures on Day One of the 6th Annual World Patient Safety, Science & Technology held in London (Photo: Business Wire) The Patient Safety Movement Foundation has taken on an inspiring and ambitious goal of reducing preventable patient deaths in hospitals to ZERO by 2020. The annual Summit in central London plays a major role in taking on that challenge with the participation of 300 global leaders, medical experts, medical technology CEOs, and patient
WuQi Technologies is Latest Licensee of CCww’s NB-IoT UE Protocol-Stack Software24.2.2018 12:06 | Tiedote
Communications Consultants Worldwide Ltd. (CCww), global innovator of 3GPP® technologies, has licensed its NB-IoT protocol-stack software to WuQi Technologies Inc., a leading global developer of highly integrated mixed-signal SoC solutions, and the latest licensee of CCww’s NB-IoT Release 13 UE protocol-stack software. Communications Consultants Worldwide (CCww) is working with WuQi Technologies for the integration of its NB-IoT protocol-stack software onto WuQi Technologies’ highly innovative SoC. This relationship, catalyzed by T2M, is enabling the production of a highly integrated, very low power, NB-IoT SoC chipset targeting IoT Smart City, Smart Home, and wearable applications. CCww’s CEO, Richard Carter, said, “NB-IoT is CCww’s latest cellular Protocol-stack SW. We are excited to be working with WuQi to enable a sector-leading NB-IoT SoC, pushing back boundaries of performance and battery life for the next generation of IoT. See our demonstration at the Mobile World Congress (Hal
IDEMIA and Octo Telematics Reinvent Car Sharing Experience by Adding Facial Recognition to EasyOpen Solution23.2.2018 18:57 | Tiedote
IDEMIA, the global leader in Augmented Identity for an increasingly digital world, announces today that it has implemented facial recognition technology on EasyOpen, a solution previously developed with its partner Omoove, a wholly-owned Octo Telematics company, Europe’s leading Shared Mobility technology provider. The digital enrolment process combined with facial recognition technology will securely enroll and authenticate the driver before EasyOpen automatically opens the car. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180223005506/en/ (Photo: Idemia) You will never rent a car the same way thanks to EasyOpen solution that combines IDEMIA’s expertise in secure service enablement and Omoove’s experience in Shared Mobility platforms and on-board technology. The first version of the solution was launched last year and allows drivers, who have already registered on the application, to use their smartphones as a key to open
SM-Optics Unveils First FLEXSET Chip, Paving the Way to Optical Micro Nodes23.2.2018 17:00 | Tiedote
SM-Optics launches FLEXSET, a single chip embedding multiple processing functionalities raising the bar for performances, density and architectural flexibility. The FLEXSET is the heart of the Lightmode product family. FLEXSET OTN switching matrix scales from 300Gbps to 1Tbps and can be addressed by ports spanning from traditional low-speed services like 2Mbps over micro-OTN, to layer2 and layer3 over ODUFlex, 100G over ODU4 and 200G over FlexO. Thanks to its embedded capabilities including multi-technology OAM, packet processing, synchronization and fast protection functions, FLEXSET operates by design at the lowest latency performances and features an Intel Stratix 10 FPGA. Following the launch of micro-OTN, a revolutionary approach to efficiently extend OTN protocol to low speed services, the launch of FLEXSET advances the vision of a highly interconnected metro network and of micro nodes. Revolving around functional block elements, micro nodes collapse in few rack units OTN and pho
Bitcoin Miner HashGains’ Crowdsale to Build Green Cloud Mining Data Centers Receives Excellent Response23.2.2018 16:56 | Tiedote
HashGains.com, a leading cryptocurrency mining platform, has launched its crowdsale program to build mega cryptocurrency cloud mining data centers in India and Canada which uses renewable energy like wind and solar as source of energy. “With the growing worries around bitcoin mining causing environmental damage, difficulty levels of mining increasing and returns heading southwards, there was no better opportunity but to build green energy data centers which run on free sources of energy like wind and solar, ensuring handsome mining rewards while taking care of environment,” said Mr Anuj Bairathi, CEO & Founder, HashGains, who is crypto enthusiast himself. HashGains is a growing cloud mining platform with more than 10,000 active customers enjoying mining returns and is expected to reach the mark of 1 million customers by 2020. To handle such a massive growth and ever growing demand of customers, it becomes even more important that mega mining centers are planned which can serve needs of
Stratecast Recognizes CARDINALITY as a “10 to Watch in 2018”23.2.2018 16:07 | Tiedote
In their new report, “Digital Transformation Means New Tools for New Business” —part of the Global Operations, Orchestration, Data Analytics, and Monetisation (ODAM) stream—Stratecast recognises CARDINALITY for their new approach towards processing big data to deliver applications such as Customer Experience, Operational Intelligence, Network Analytics, Churn Management and Marketing Intelligence. Stratecast identified that the CARDINALITY Perception Platform brings large amounts of data from multiple CSP sources together in order to address a growing group of data analysis needs from different operational work teams and the wider business. It also does this at ingestion rates and storage levels previously thought not practical. "Using design principals tied to microservices and containerized solution components, the platform is a major advancement in meeting CSP data analysis needs at a price untouchable by previously-defined platforms," said Karl Whitelock, Stratecast director of glo
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