CHMP Grants Positive Opinion for KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in Combination With KALYDECO ® (ivacaftor) in People Ages 12 and Older With Cystic Fibrosis With the Most Common Genotypes
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) 150 mg to treat people with cystic fibrosis (CF) ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. If approved, up to 10,000 people in Europe with CF ages 12 and older who have one F508del mutation and one minimal function mutation would be newly eligible for a medicine that targets the disease- causing protein defect. Additionally, people 12 years of age and older who have two F508del mutations and who are currently eligible for one of Vertex’s other EMA-approved cystic fibrosis medicines would be eligible for the new triple combination regimen.
The CHMP positive opinion was based on the results of two global Phase 3 studies in people with CF: a 24-week placebo-controlled study in people ages 12 years and older with one F508del mutation and one minimal function mutation and a 4-week head-to-head study of the triple combination therapy in comparison to tezacaftor/ivacaftor in people ages 12 years and older with two F508del mutations. Both Phase 3 studies showed statistically significant and clinically meaningful improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated.
“The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 12 years and older with an F/F or F/MF genotype are unprecedented. In addition to improvements in lung function, the data have shown improvements in multiple important outcome measures, including quality of life as measured by the CFQ-R respiratory domain score,” said Professor Marcus A. Mall, M.D., Head of Department of Pediatric Pulmonology, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. “Both the clinical and patient communities are excited that more people with CF will be able to benefit from CFTR modulators.”
“We are delighted to have received this positive opinion from CHMP. If approved, this would be the first CFTR modulator for people with one F508del mutation and one minimal function mutation and would bring additional benefit to people with two F508del mutations,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “This milestone brings us one step closer to delivering this innovative CF medicine to those who are waiting, and toward our ultimate goal of providing a therapeutic option for every person with this rare and devastating disease.”
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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With KALYDECO® (ivacaftor)
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) is an investigational medicine developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The EU submission for KAFTRIO® was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).
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, 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, statements made by Dr. Mall and Dr. Bozic in this press release, and statements regarding our expectations for the approval of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) in Europe, and our expectations regarding the eligible patient population 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 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, risks related to obtaining approval for and commercializing medicines in Europe, and other risks listed under Risk Factors in Vertex's annual report and subsequent 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
International: +44 20 3204 5275
U.S.: +1 617-341-6992
About Business Wire
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
US-Cable Operator Armstrong Turns To Verimatrix To Streamline & Secure VOD Launch28.9.2020 07:15:00 CEST | Press release
Regulatory News: Verimatrix, (Paris:VMX) (Euronext Paris: VMX), the leader in powering the modern connected world with people-centered security, today announced that Butler, Pennsylvania-based Armstrong, a leading U.S. cable company offering service in more than 469 zip codes, deployed the Verimatrix Video Content Authority System (VCAS™). Founded 74-years ago, Armstrong is committed to securely offering a wide array of popular streaming options for its large customer base. Armstrong recently incorporated a more sophisticated TiVo platform into its set-top boxes in order to remain at the cutting-edge of technological changes in the television industry. In order to make the transition worthwhile, the company required a high-quality, easy-to-integrate encryption solution that would allow it to offer as many streaming options – and it selected the Verimatrix VCAS solution. “Their high-level anti-piracy software is widely accepted by content owners, something other encryption services simp
Spirit of Wipro Run Brings Together Thousands of Participants Globally27.9.2020 08:52:00 CEST | Press release
Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today organized the 15th edition of the ‘Spirit of Wipro’ (SOW) Run globally with thousands of participants across 35 countries, running separately, yet together in spirit, at a time of their choosing. The theme for this year’s Run was “United. Unstoppable”, which celebrated camaraderie, resilience and a shared sense of community among several thousands of Wipro employees, their friends and families, alumni, customers, partners and suppliers. The SOW Run is an annual, global tradition at Wipro and has been held every year since its inception. This year too, the Run was organized on schedule despite the pandemic induced constraints. The runners strictly adhered to the local COVID-19 guidelines and safety protocols in various countries while participating in the event. Commenting on the Run, Thierry Delaporte, Chief Executive Officer and Managing
BCG Collaborates with Tencent Marketing Insight to Release 2020 BCG x Tencent Digital Luxury Report25.9.2020 12:21:00 CEST | Press release
Since early 2020 when the COVID-19 pandemic broke out worldwide, the luxury market has been hit hard and is expected to decline 25% to 45% compared to the previous year. The Chinese luxury market, however, which has benefitted from successful domestic control of the pandemic and has taken the lead in recovery against a depressed global market environment, is forecast to grow from 20% to 30% in the whole year of 2020. Thanks to its stunning market performance, China has become the main battleground of each major luxury brand. In was in this environment that Boston Consulting Group (BCG) joined hands with Tencent Marketing Insight (TMI) to release the 2020 BCG x Tencent Digital Luxury Report, drawing on deep insights into post COVID-19 trends in the luxury market and consumers based on BCG and TMI’s quantitative consumer research and industry experience, as well as TMI’s marketing and big data capabilities. Crystal Hao, Managing Director & Partner of BCG said, “The luxury market in China
U.S. MRTP Authorization Pathway Explored in PMI’s Latest Scientific Update25.9.2020 12:15:00 CEST | Press release
Philip Morris International (PMI) (NYSE: PM) has released the 11th edition of its Scientific Update, a regularly issued publication on its research efforts to develop and scientifically assess a range of smoke-free alternatives to cigarettes. The latest edition features an overview of the historic decision made by the U.S. Food and Drug Administration (FDA) to authorize the marketing of the IQOS Tobacco Heating System with reduced exposure information. The Scientific Update explains the various MRTP decisions under U.S. law; the steps, processes, and timeline PMI undertook; and the ongoing post-market surveillance and studies that will monitor the impact of the orders on consumer perceptions, behavior, and health. “The FDA undertook a scientific and regulatory evaluation of IQOS and determined that it is ‘appropriate for the promotion of public health’” said Dr. Gizelle Baker, director of Global Scientific Engagement at PMI. “In this edition of our Scientific Update, we describe how th
Palladio Completes $20 Million Series B Financing25.9.2020 12:09:00 CEST | Press release
Palladio Biosciences, Inc. (Palladio), a privately-held, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney, announced today the completion of a $20 million Series B investment. The financing was led by new investor, Samsara BioCapital, with participation from new investor, the Roche Venture Fund and existing investors, Medicxi and Osage University Partners. Proceeds from the financing will fund Palladio’s Phase 3 ALERT Study and advance operations. Srinivas Akkaraju M.D, Ph.D, Managing General Partner of Samsara BioCapital, stated, "We are very excited about our investment in Palladio to support the development of lixivaptan, a drug that has the potential to deliver a meaningful therapeutic advancement in the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). The ALERT Study is an important step in assessing the safety differential of lixivaptan.” “We are very pleased that Samsara and the Roche Venture Fund recognize the opp
Ant Group Launches “Trusple,” an AntChain-Powered Global Trade and Financial Services Platform for SMEs and Financial Institutions25.9.2020 09:54:00 CEST | Press release
Ant Group, a leading provider in the development of open platforms for technology-driven inclusive financial services, and the parent company of China’s largest digital payment platform Alipay, today unveiled Trusple, an international trade and financial service platform powered by AntChain, the company’s blockchain-based technology solutions. Trusple aims to make it easier and less costly for all participants – especially Small-to-Medium Enterprises (SMEs) – to sell their products and services to customers around the world. It also reduces costs for financial institutions so they can better serve SMEs in need. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200925005110/en/ (Photo: Business Wire) Based on the concept of “Trust Made Simple,” Trusple works by generating a smart contract once a buyer and a seller upload a trading order on the platform. As the order is executed, the smart contract is automatically updated with k
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