Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
“Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older. With today’s approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “Today’s approval is a testament to our commitment to keep going until all people with CF have a treatment option.”
The label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.
KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.
KALYDECO® (ivacaftor) is already approved in Europe for people with CF ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells 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 early 30s.
About KALYDECO® (ivacaftor)
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.
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 diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
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, statements made by Dr. Reshma Kewalramani in this press release, and statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to KALYDECO for eligible patients four months of age and older across countries in Europe, and our plans to secure access to KALYDECO for additional eligible patients four months of age and older in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Pharmaceuticals Incorporated
International: +44 20 3204 5275
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Rand Technology Appoints Key Executives Across Global Regions4.12.2020 02:30:00 EET | Press release
Rand Technology (Rand) has appointed Kim Fix, Chief Operating Officer (COO), Americas & Europe; Frederick Fu, President and Managing Director, Asia-Pacific (APAC); Nami Mokri, Vice President, Sales & Sourcing, APAC; Kevin Sheehan, Chief Information Officer (CIO); and Jennifer Strawn, Director, Sourcing & Procurement, Americas & Europe. Kim Fix, Global Vice President, QA & Operations, has been appointed to COO, Americas & Europe. Kim will lead Rand’s sales and sourcing teams’ operations across the Americas and Europe and oversee global compliance, quality assurance, and operations. Kim has been integral to Rand’s preferred status with its business partners and multi-site ISO, OHSAS, ANSI ESD, and R2 certifications. Frederick Fu joined Rand as President and Managing Director, APAC. He previously served as Avnet’s President of APAC. He has been fundamental to Rand’s efficacy, growth, strategy, and success in the APAC region. Nami Mokri was appointed Vice President, Sales & Sourcing, APAC.
Kalray Joins MLCommons as Founding Member3.12.2020 20:30:00 EET | Press release
Kalray (Euronext Growth Paris: FR0010722819 – ALKAL) (Paris:ALKAL), a pioneer in processors for new intelligent systems, today announced it has joined MLCommons as founding member. MLCommons is a not for profit, engineering consortium founded by 30+ global technology and academic leaders in AI and Machine Learning to accelerate machine learning innovation and broaden access to this critical technology for the public good. Initially formed as MLPerf, MLCommons boasts a founding board that includes representatives from Alibaba, Facebook AI, Google, Intel, NVIDIA and Professor Vijay Janapa Reddi of Harvard University with the objective to deliver industry-wide benchmarks, best practices and datasets to speed computer vision, natural language processing, and speech recognition development for all. “MLCommons has a clear mission - accelerate Machine Learning innovation to ‘raise all boats’ and increase its positive impact on society” said Peter Mattson, President of MLCommons. “We are excit
Hyperloop Transportation Technologies Accelerates with 100 Engineers from World Leading Firm Altran3.12.2020 19:40:00 EET | Press release
Hyperloop Transportation Technologies (HyperloopTT) announced today an agreement with world-leading engineering and R&D services provider Altran, part of the Capgemini Group. The agreement includes one hundred Altran engineers to accelerate the development of commercialized passenger hyperloop systems at the HyperloopTT facility in Toulouse, France, the Aerospace Valley of Europe. HyperloopTT’s European R&D Center is the home of the world’s only full-scale hyperloop system, which has been undergoing testing and optimization since 2019. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201203005174/en/ HyperloopTT's full-scale test system in Toulouse, France (Photo: Business Wire) The agreement advances HyperloopTT’s capabilities in a lean way, negotiated within HyperloopTT’s hybrid collaborative framework. The announcement comes after recent commitments from both the European Union and the United States, emphasizing the develop
Dan Bilzerian Joins Team GGPoker & Celebrates With $100,000 Birthday Freeroll3.12.2020 19:00:00 EET | Press release
GGPoker today announces that Dan Bilzerian has joined Team GGPoker as a brand ambassador. One of the world’s most recognizable poker players and social media influencers, with over 32 million followers on Instagram, “Blitz” will represent GGPoker live and online. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201203005724/en/ Dan Bilzerian, GGPoker's newest brand ambassador. (Photo: Business Wire) To celebrate the news, the online poker room will host a special 40th birthday party for the ‘King of Instagram’ on December 7 in the form of a $100,000 Birthday Freeroll poker tournament. This free-to-enter poker game is open to all new GGPoker players that sign up using code BLITZ40 and deposit $10 or more. What : Dan’s $100,000 Birthday Freeroll When : Monday, December 7 at 17:00 UTC How To Enter : Sign up using code BLITZ40, deposit at least $10 and use your ticket to register for the freeroll tournament Tournament Prize Pool :
Odense Investor Summit Introduces Investors to 15 Promising Robot Companies in Europe’s Robot Capital3.12.2020 18:47:00 EET | Press release
In just five years, robotic start-ups based in Odense, Denmark – the robot capital of Europe – have attracted more than 976 million USD. This year, the local Danish entrepreneurs will be joined by companies from the RobotUnion Super Stars; finalists from the pan-European robot acceleration program. The event is being sponsored by Vækstfonden, the Danish State’s investment fund, with the objective of making matches between leading Danish entrepreneurs seeking capital backing, and top international investors on the hunt for the next “golden goose” in the world of robotics. On December 9th, a carefully selected field of robot enterprises seeking capital will be presenting their ground-breaking automation solutions for a broad spectrum of industries at this year’s Odense Investor Summit. The 2020 summit is being held as a virtual event to involve participants from all parts of Europe and provide access to a new audience while still observing the prevailing COVID-19 restrictions. In additio
IEEE Launches 802.3™ Beyond 400Gb/s Ethernet Study Group to Define Next High Speed Threshold for Ethernet Data Rate3.12.2020 18:30:00 EET | Press release
IEEE, the world's largest technical professional organization dedicated to advancing technology for humanity, and IEEE Standards Association (IEEE SA) today announced the formation of a study group within the IEEE 802.3™ Ethernet Working Group. With bandwidth demand growing across all areas of Ethernet connectivity globally, the IEEE 802.3 Beyond 400Gb/s Ethernet Study Group will explore initiating a new IEEE project that would standardize capabilities exceeding today’s maximum data rate of 400 Gigabit per second (Gb/s) for IEEE 802.3, IEEE Standard for Ethernet. The publicly available IEEE 802.3™ Ethernet Bandwidth Assessment (BWA) report, released in April 2020, showed that trends around traffic drivers such as video, hyperscale data centers, 5G and deployment of IEEE 802.11™ “Wi-Fi®” all point toward an ongoing bandwidth explosion for Ethernet networks around the world in the years ahead. Undeniably, simultaneous trends point toward more users, faster access speeds, more services, m
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom