Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) in Infants With Cystic Fibrosis Ages 6 Months to Less Than 12 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 infants with cystic fibrosis (CF) ages 6 months to less than 12 months weighing 5 kg and more 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.
“Today’s approval is another milestone in our commitment to treat all people with CF as early in life as possible, given manifestations of CF are often present at birth,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “For the first time, children with CF in Europe as young as 6 months with certain mutations now have access to a medicine that treats the underlying cause of their disease.”
The label update is based on data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF who are less than 24 months of age and have a CFTR gating mutation. The study showed a safety profile similar to that observed in previous Phase 3 studies of older children and adults, and improvements in sweat chloride, a secondary endpoint.
KALYDECO® (ivacaftor) is already approved in Europe for the treatment of CF in patients ages 12 months and older who have one of the 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 18 years and older who have the R117H mutation in the CFTR gene.
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.
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 diseases. The company has four approved medicines in the U.S. and three approved medicines in Europe 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 disease. 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, UK. 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
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, the statement in the second paragraph of the press release. 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 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 data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and 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, +1-617-341-6108
Zach Barber, +1-617-341-6470
Leah Gibson, +1-617-961-1507 or
Sarah D'Souza, +1-617-341-6992
Europe & Australia:
Patricia Dessert, +44 7543 237825
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
Iguazio Raises $24M to Accelerate Growth and Global Penetration of its Data Science Platform27.1.2020 15:00:00 EET | Press release
Iguazio, the data science platform for real time machine learning applications, today announced that it has raised $24M of funding. The round was led by INCapital Ventures, with participation from existing and new investors, including Pitango, Verizon Ventures, Magma Venture Partners, Samsung SDS, Kensington Capital Partners, Plaza Ventures and Silverton Capital Ventures. The funds will be used by Iguazio to accelerate its growth and expand the reach of its data science platform to new global markets. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200127005301/en/ Iguazio co-founders Yaron Segev (left), Yaron Haviv, and Asaf Somekh. Photo: Yanai Yechiel The demand for AI applications is on the rise. According to Gartner, AI augmentation alone will create $2.9 trillion of business value in 2021. However, there are still many challenges in deploying AI solutions in an effective and scalable way. An estimated 87% of data scienc
Iguazio Deployed by Payoneer to Prevent Fraud with Real-time Machine Learning27.1.2020 15:00:00 EET | Press release
Iguazio, the data science platform for real time machine learning applications, today announced that Payoneer, the digital payment platform empowering businesses around the world to grow globally, has selected Iguazio’s platform to provide its 4 million customers with a safer payment experience. By deploying Iguazio, Payoneer moved from a reactive fraud detection method to proactive prevention with real-time machine learning and predictive analytics. Payoneer overcomes the challenge of detecting fraud within complex networks with sophisticated algorithms tracking multiple parameters, including account creation times and name changes. However, prior to using Iguazio, fraud was detected retroactively, enabling customers to only block users after damage had already been done. Payoneer is now able to take the same sophisticated machine learning models built offline and serve them in real-time against fresh data. This ensures immediate prevention of fraud and money laundering with predictiv
Oasis Smart SIM Achieves Certification of HELIOS eUICC in Accordance With GSMA M2M Specifications27.1.2020 14:48:00 EET | Press release
Oasis’ newly certified HELIOS eUICC Operating System can be used in any type of mobile device, whilst guaranteeing total security and interoperability with the rest of the eSIM ecosystem. This certification was done by an independent accredited laboratory following the GSMA SGP.16 v1.0 validation requirements and processes. Rigorous testing of HELIOS eUICC by a third-party lab verified the compliance with GSMA specifications and standard interoperability. The goal is to ensure that mobile network operators are able to use HELIOS eUICC in complete security and in a reliable manner. To address the market needs, HELIOS eUICC is available both as a software-only Operating System or preloaded into Secure Elements from leading semiconductor companies. Its complete interoperability ensures that HELIOS eUICC can be remotely managed by any Subscription Manager compliant with the GSMA specifications. Patrick Cao, Chief Operating Officer, said “We are proud to obtain this certification. This conf
A Better Alternative to Banking - ClubSwan.com27.1.2020 12:00:00 EET | Press release
ClubSwan.com, the leader in membership driven financial solutions, further expands its innovative alternative banking capabilities to key global markets. As traditional financial institutions continually struggle to provide innovative and meaningful ways to bring value to individual and corporate customers and as digital assets/crypto currencies are experiencing global growth, ClubSwan.com continues to evolve to meet the efficiencies and demands that these new assets and our customers require and to which traditional banks have no answer. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200127005046/en/ Instead, closed bank accounts, rejected transactions, crippling delays with customers receiving their funds (if at all) are all the norm with traditional banks. To add to this dilemma, the availability of trustworthy solutions where customers have peace of mind for both crypto assets and traditional currencies have been non-exi
GSMA Recognizes Turkcell and P.I. Works for the Value of AI Based Network Automation27.1.2020 10:00:00 EET | Press release
Turkcell and P.I. Works have been recognized by GSMA Future Networks Programme for achieving exceptional efficiency gains in network operations and ensuring superior customer experience. P.I. Works and Turkcell have successfully shown that the AI based, service aware network automation delivered stronger improvements as compared to conventional automation solutions. The details of the deployed solutions and their benefits are provided in a joint white paper authored by GSMA, Turkcell and P.I. Works under GSMA’s Future Networks Programme. To read the white paper please visit: https://www.gsma.com/futurenetworks/wiki/case-study-ai-use-cases-in-service-assurance/ GSMA Future Networks Programme intends to help operators share best practices and discover new opportunities that will enable them to reduce operational and capital expenditures. Given the success of the project, GSMA invited Turkcell and P.I. Works to join the program and provide insights for the AI-based, Service Aware Automate
Phase 2 DESTINY-Gastric01 Trial of DS-8201 Versus Chemotherapy Met Primary Endpoint27.1.2020 10:00:00 EET | Press release
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca today announced positive topline results from the pivotal phase 2 DESTINY-Gastric01 trial of DS-8201, a HER2 directed antibody drug conjugate (ADC), in patients with HER2 positive unresectable or metastatic gastric or gastroesophageal junction cancer that had progressed following two or more treatment regimens including trastuzumab and chemotherapy. The trial met its primary endpoint, achieving a statistically significant and clinically meaningful improvement in objective response rate (ORR), as assessed by an independent review committee, in patients treated with DS-8201 versus investigator’s choice of chemotherapy (irinotecan or paclitaxel monotherapy). DS-8201 also showed a statistically significant and clinically meaningful improvement in overall survival (OS), a key secondary endpoint. The safety profile observed for DS-8201 in DESTINY-Gastric01 was consistent with previous clinical trials. These results c
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