Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) to Treat Patients with Cystic Fibrosis Aged 12 to <24 months with Certain Mutations in the CFTR Gene
Vertex Pharmaceuticals (Europe) Limited today announced that the European Commission has granted approval of the label extension 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.
“For the first time, EU physicians can now treat the underlying cause of CF earlier than ever, helping to improve clinical outcomes in children as young as 12 months,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We look forward to supporting additional research on the potential benefit of early intervention with our medicines, with the goal of bringing treatment to all people living with CF.”
The label update is based on data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF aged 12 to <24 months who have one of 10 mutations in the CFTR gene that demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults, and improvements in sweat chloride, a key secondary efficacy endpoint.
Ivacaftor is already approved in Europe for the treatment of CF in patients aged two years and older who have one of the nine following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. It is also approved for the treatment of CF in patients aged 18 years and older who have an R117H mutation in the CFTR gene.
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 the ARRIVAL Study
The ARRIVAL study is an ongoing Phase 3 open-label safety study of 25 children with CF aged 12 to <24 months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H; patients with the R117H mutation were eligible to enroll in regions where ivacaftor is approved for use in patients 2 through 5 years of age with an R117H mutation). The study demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued due to adverse events. Treatment was interrupted in two patients who had elevated liver enzymes greater than eight times the upper limit of normal, but continued to receive ivacaftor after a dose interruption. The most common adverse events (≥30%) were cough (74%), pyrexia (37%), elevated aspartate aminotransferase (37%), elevated alanine aminotransferase (32%) and runny nose (32%). Four serious adverse events were observed in two patients.
Mean baseline sweat chloride for the children in this study was 104.1 mmol/L (n=14). Following 24 weeks of treatment with ivacaftor, the mean sweat chloride level was 33.8 mmol/L (n=14). In the 10 subjects with paired sweat chloride samples at baseline and week 24, there was a mean absolute change of -73.5 mmol/L. Sweat chloride is used as a tool to diagnose infants with CF, where levels greater than or equal to 60 mmol/L indicate that CF is likely, levels of 30-59 mmol/L indicate CF is possible and levels less than 30 indicate that CF is unlikely. These data were presented at the 41st European Cystic Fibrosis Society (ECFS) Conference in June 2018 and published in The Lancet Respiratory Medicine (Volume 6, No 7, July 2018).
Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
People with CF who have specific mutations in the CFTR gene are currently benefiting from ivacaftor in countries across North America, Europe and in Australia.
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, Australia and Latin America. 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 nine years in a row.
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. Kewalramani’s statement in the second paragraph of this press release. 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, risks related to commercializing KALYDECO® for people with cystic fibrosis aged 12 to <24 months and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Eric Rojas, +1-617-961-7205
Zach Barber, +1-617-341-6470 or
Sarah D'Souza, + 1-617-341-6341
Europe & Australia:
Marie von Seyfried, + 44 7933 500887
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
Age Matters: PMI Calls All Tobacco and E-Cigarette Companies To Do Their Part to Guard Against Youth Nicotine Use23.4.2019 20:29:00 EEST | Tiedote
Philip Morris International Inc. (PMI) (NYSE: PM) today reaffirmed its vision for the future: To achieve a smoke-free world; that is, one without cigarettes. For the hundreds of millions of men and women globally who will otherwise continue to smoke, PMI’s goal is to switch them to nicotine-containing products that are scientifically substantiated as better choices than continuing to smoke. Our strong belief—if you don’t smoke, don’t start—begins with a continued and robust focus on preventing youth from beginning to smoke or use nicotine products. “Given the rapid pace of innovation in the tobacco products space, we are emphatic that youth should not use any tobacco- or nicotine-containing product. Nicotine is addictive. It is not risk-free, and it poses particular risks for adolescents. We know that great care must be taken to achieve our smoke-free goal. Youth should not become nicotine users. Former smokers and never smokers should not return to, or pick up, the tobacco or nicotine
Personalis, Inc. to Present at European NeoAG Summit 201923.4.2019 18:00:00 EEST | Tiedote
Personalis, Inc., a leader in advanced genomics for cancer, today announced that the company will present at European NeoAG Summit 2019 in Amsterdam on April 24th at 2:30 PM CEST. The presentation, entitled “ImmunoID NeXT Platform: Improving Neoantigen Prediction, TME Assessment, and ctDNA Detection for Vaccine Development,” will introduce Personalis’ new universal cancer immunogenomics platform, ImmunoID NeXT™. In addition to an overview, the presentation will highlight innovative genomics methods for more comprehensive assessment of neoantigens, which include improving neoantigen-to-MHC binding prediction and ranking and assessing TME/TCR and tumor escape mechanisms that may impact vaccine response. ImmunoID NeXT is the first platform to provide comprehensive analysis of both a tumor and its immune microenvironment from a single sample. The platform can be used to investigate the key tumor- and immune-related areas of cancer biology, consolidating multiple oncology biomarker assays i
Keio Plaza Hotel Tama Opens My Melody, Little Twin Stars Rooms on June 15, 2019 - Sweet Dreams in Our Hello Kitty and Sanrio Character Rooms23.4.2019 17:05:00 EEST | Tiedote
The Keio Plaza Hotel Co., Ltd., one of Japan’s most prestigious hotel companies operating hotels located in the Shinjuku district of Tokyo and other locations, is proud to announce the strengthening of its collaborative relationship with Sanrio Co., Ltd. (Headquarters: Shinagawa, Tokyo, President and CEO: Shintaro Tsuji) to expand facilities and services featuring “Hello Kitty” and other Sanrio characters at our Keio Plaza Hotel Tama. Our Tama Hotel will renovate a total of four additional rooms, including two rooms each in the themes of “My Melody” and “Little Twin Stars” (Kiki and Lala) who have been adored by fans around the world since their creation in 1975, to be opened on Saturday June 15, 2019, and thereby become the world’s first hotel to operate rooms in these themes on a regular basis. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190423005311/en/ New Sanrio characters rooms, including two rooms each in the theme
Project Management Institute, Inc. (PMI) Delivers Full Suite of Certification Exams in Over 5,000 Locations Through Expanded Testing Agreement with Pearson VUE23.4.2019 17:00:00 EEST | Tiedote
Project Management Institute, Inc. (PMI), the leading not-for-profit professional membership association for the project management profession, and Pearson VUE, a global leader in the computer-based testing industry, have announced an expanded, multi-year agreement that will offer more testing options for candidates looking to earn PMI certifications at all stages of their career. The new agreement will offer candidates access to even more PMI certification tests as well as more flexibility in their testing location options, thanks to Pearson VUE’s global network of testing centers and online proctoring solutions. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190423005149/en/ Beginning July 1, 2019, seven additional PMI programs, including the Project Management Professional (PMP)® – renowned as the global gold standard for project management professionals – will become available to professionals around the globe through Pe
Andersen Global Marks Growth to 50 Countries with Collaborating Firm in Zambia23.4.2019 16:30:00 EEST | Tiedote
Andersen Global announced it achieved a new milestone today in its continuing growth with the addition of Mulenga Mundashi Kasonde Legal Practitioners (MMKLP). MMKLP, a law firm in Zambia, has signed a Collaboration Agreement with Andersen Global. The international association now has presence in 50 countries through its member and collaborating firms. Led by five partners, MMK has been advising clients in corporate mergers and acquisitions, tax, litigation, and labor and industrial resolutions since 1999. The firm is now one of the largest law firms in Zambia in its twentieth year. The collaborating firm will be the eleventh location in Africa for Andersen Global. “We’ve had the opportunity to advise on some of the highest profile financial events for Zambia in modern history,” said Micheal Mundashi, MMKLP Managing Partner. “One of the things that keeps us moving forward is the desire to provide even more for our local and international clients. Collaborating with Andersen Global will
Aventri Introduces Uber Vouchers to Deliver New Transportation Options23.4.2019 16:00:00 EEST | Tiedote
Event management software (EMS) leader Aventri today announces a new partnership with Uber for Business. Aventri is the first EMS provider to launch a partnership with Uber for Business to offer customers the opportunity to utilize Uber Vouchers. Uber Vouchers’ web-based tool allows event organizers to provide VIP treatment to attendees with stress-free, reliable transportation to and from events. “We’re always looking for new partners to help our customers streamline events and add value,” said Brad Langley, vice president of channel and partner management for Aventri. “Uber for Business fits the bill. Using Uber Vouchers, organizers spend less time arranging logistics for moving groups, and their guests can come and go at a time that’s convenient for them.” Using Uber Vouchers, organizers can enhance the journey for themselves and their guests. Some benefits include: Customized Control: Organizers can easily create and manage campaigns online by setting parameters for pickup, drop-of
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