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
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
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
Ingenic Semiconductor Integrates Allegro DVT Encoding IP into Next-Generation Smart-Video System-On-Chip Solutions3.7.2020 12:46:00 CEST | Press release
Allegro DVT, the leading provider of video semiconductor IP solutions, today announced that Ingenic Semiconductor, one of the leading system-on-chip companies in China has integrated the AL-E150 high-performance video encoding IP into its latest generation of Smart Video products. The Smart Video product family from Ingenic Semiconductor features highly integrated Video Processor system-on-chip (SoC) solutions targeted at various video applications such as consumer and professional video surveillance and battery-operated cameras. One of Ingenic latest video processors, includes Allegro DVT’s AL-E150 H.264/HEVC/JPEG encoder to handle compression of multiple video channels at resolutions up to 5Mpixels. This chip is now shipping in mass-volume. Brad Liu, VP of Smart Video BU, Ingenic commented, “With the AL-E150,Allegro DVT is able to offer a multi-format and feature-rich encoder IP which perfectly meets our requirements and represents the best trade-off between silicon area, high-video
Imaweb-IDF Group Completes Its Acquisition of Serinfer3.7.2020 11:16:00 CEST | Press release
Imaweb/IDF Group (“The Group”), one of the largest developers of customer relationship management (“CRM”) solutions and dealership management systems (“DMS”) for the automotive industry in Europe, announced today that it has completed the acquisition of Serinfer Servicios Informáticos S.L. (“Serinfer”), the computer software segment of the Alvariño Group, a Spanish developer, distributor and supplier of DMS for automobile dealers. The Group is backed by Providence Strategic Growth (“PSG”), the growth equity affiliate of premier asset management firm Providence Equity Partners (“Providence”). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200703005051/en/ Serinfer develops a range of sophisticated products to address the evolving needs of automobile dealers. Its flagship offering is SERAUTO, a complete DMS solution that works modularly and can also be integrated to the existing technology of any dealership or workshop. SERAUT
Taiwan’s COVID-19 response win applause, friendship from around the world3.7.2020 10:52:00 CEST | Press release
Once considered the most vulnerable to the COVID-19 outbreak because it’s so close to China, Taiwan has turned out to be a model for the world’s fight against the pandemic, thanks to quick decisions and early measures by its government, and to the high standard of the island nation’s public health system and its people. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200703005042/en/ The management of Grand Hotel in Taipei asks its guests to help form Zero on the building’s facade to celebrate no new COVID-19 cases for the sixth day in a row on May 1, 2020. (Hsu Chao-chang, CNA) According to real-time data released on Worldometer (https://www.worldometers.info/coronavirus/), a website tracking global COVID-19 figures, as of June 30, Taiwan, with only 447 cases, ranked 155th among 215 countries in terms of the number of infections. Only three entities with fewer cases have a larger population than Taiwan’s, which currently sta
Northern Data announces new Block.one backed customer3.7.2020 08:27:00 CEST | Press release
Northern Data AG (XETRA: NB2, ISIN: DE000A0SMU87), one of the world's largest providers of high-performance computing (HPC) solutions, continues to enjoy a very successful 2020 with the signing of 180 MW of capacity with a new customer. Northern Data's most recent customer is a Bitcoin mining operation backed by Blockchain Powerhouse Block.one with an initial 180 MW volume and an upsize option to 300 MW, which the company signed today and which shall go live in early 2021. Brendan Blumer, CEO of Block.one, says: "Block.one's focus on leveraging EOSIO to bring increased scalability and transparency to the greater blockchain industry led us to Northern Data and their strategic market positioning and strong leadership. We're excited to be collaborating with them to introduce unprecedented Bitcoin mining solutions, powered by EOSIO." Aroosh Thillainathan, CEO of Northern Data AG adds, "We are pleased that Block.one is convinced of our services and that we have won the company as another bl
ViiV Healthcare Announces US FDA Approval for Rukobia (fostemsavir), a First-in-Class Treatment for HIV in Adults With Few Treatment Options Available2.7.2020 22:39:00 CEST | Press release
ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. and Shionogi Limited as shareholders, today announced that the US Food and Drug Administration (FDA) has approved Rukobia (fostemsavir), 600 mg extended-release tablets. Rukobia is a novel attachment inhibitor for the treatment of HIV-1 infection indicated for use in combination with other antiretroviral (ARV) therapies in heavily treatment-experienced (HTE) adults with multidrug-resistant HIV-1 infection, who are failing their current ARV regimen due to resistance, intolerance or safety considerations. Significant advances over the past few decades have dramatically improved HIV treatment and for many, HIV is considered a manageable life-long condition. However, HTE adults – which account for approximately 6% of adults living with HIV who are on treatment – have little to no options left due to resistance, tolerability or safety considerations.1 HTE adults are at risk of progressing to AIDS and
Vertex Files Registration Statement for Proposed Initial Public Offering2.7.2020 22:31:00 CEST | Press release
Vertex, Inc.,a leading provider of tax technology and services, today announced that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (SEC) relating to a proposed initial public offering of its common stock. The number of shares to be offered and the price range for the proposed offering have not yet been determined. Vertex intends to list its common stock on the NASDAQ Global Market under the ticker symbol “VERX.” Goldman Sachs & Co. LLC and Morgan Stanley are acting as joint lead book-running managers and as representatives of the underwriters for the proposed offering. BofA Securities, Citigroup and Jefferies are also acting as book-running managers and JMP Securities, Stifel, William Blair and CastleOak Securities, L.P. are acting as co-managers for the proposed offering. The proposed offering will be made only by means of a prospectus. When available, copies of the preliminary prospectus relating to these securities may be obtained