Business Wire

Vertex Announces European Commission Approval for KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis Patients 12 Years and Older With At Least One F508del Mutation in the CFTR gene

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With this extension of the indication, CF patients 12 years and older who are heterozygous for the F508del-CFTR mutation and a residual function (F/RF) or gating mutation (F/G) are eligible for the triple combination therapy for the first time, and the majority of people ages 12 years and older with CF in Europe are now eligible for KAFTRIO.

“This indication extension is important as CF patients in Europe with gating and residual function mutations will have access to KAFTRIO for the first time,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “We look forward to working with health authorities and governments to ensure that all eligible patients who can benefit from this medicine have access as soon as possible.”

In both the United States and Australia, where the triple combination therapy is known as TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), it is already licensed for the treatment of people with CF ages 12 years and older who have at least one copy of the F508del mutation in the CFTR gene, regardless of the other mutation type.

“In clinical trials ivacaftor/tezacaftor/elexacaftor plus ivacaftor showed positive significant results in people with cystic fibrosis ages 12 years and older who have at least one copy of the most common gene mutation (F508del) with the latest results showing clinical benefits in individuals who have an additional ‘gating’ (F/G) or ‘residual function’ (F/RF) gene mutation. It is welcome news for the CF community that the European regulatory authority has extended the licensed indications for this therapy based on the latest trial results which means that additional patients will gain access to this medication,” said Dr Peter Barry, Honorary Senior Lecturer at The University of Manchester.

As a result of long-term reimbursement agreements in the Republic of Ireland, Northern Ireland, Denmark and Luxembourg, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for the triple combination regimen shortly following regulatory approval by the European Commission.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally.1 CF is a progressive, multi-system disease that affects the lungs, liver, gastrointestinal (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 With Ivacaftor

KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was 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. Ivacaftor/tezacaftor/elexacaftor is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The latest approved EU licensed indication for ivacaftor/tezacaftor/elexacaftor was supported by positive results of three global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study (Study 445-102) in 403 people with one F508del mutation and one minimal function mutation (F/MF), a four-week Phase 3 study (Study 445-103) in 107 people with two F508del mutations (F/F), and a Phase 3 study (Study 445-104) in 258 people heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF).2

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

About Vertex

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 cell and genetic 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.

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 and Dr. Peter Barry in this press release, statements regarding the eligible patient population in Europe and our expectations regarding the timing of access to KAFTRIO® in combination with ivacaftor for eligible patients ages 12 years 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 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 commercializing KAFTRIO® in combination with ivacaftor in Europe, and other risks listed under the heading “Risk Factors” in Vertex's annual report filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

1 Vertex. Data on File. Global CF EPI. 2021.
2 EMA. Kaftrio Summary of product characteristics.Vertex Pharmaceuticals (Ireland) Limited. Available at: https://www.ema.europa.eu/en/documents/product-information/kaftrio-epar-product-information_en.pdf
Last accessed April 2021.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or
617-961-7163

Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: 617-341-6992

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(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

SentinelOne Receives Highest Score for the Type C Use Case in the Gartner 2021 Critical Capabilities for Endpoint Protection Platforms14.5.2021 16:00:00 CEST | Press release

SentinelOne, the autonomous cybersecurity platform company, today announced that Gartner has positioned SentinelOne with the highest score in customer use case C in Gartner’s 2021 Critical Capabilities for Endpoint Protection Platforms report1. SentinelOne also received the highest score across all three Critical Capabilities use cases out of the 19 vendors included in the report. According to Gartner, “Type C organizations typically view technology as an expense or operational necessity and use it as a means to reduce costs.” Additionally, Gartner comments, “For EPP, these organizations focus on prevention, rather than on integrated detection and response capabilities and solutions that offer a complement of managed services.” “Recognition for this use case is a testament to SentinelOne’s ability to offer cost-effective and efficient solutions for enterprises of all types, including those who need to prioritize innovation and capabilities with budget and operational constraints,” said

Wipro Limited Appoints Tulsi Naidu to Its Board14.5.2021 14:22:00 CEST | Press release

Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today announced the appointment of Ms. Tulsi Naidu, a widely respected business leader, to its Board of Directors for five years effective July 1, 2021, subject to the approval of shareholders. Tulsi Naidu will serve as an Independent Director on the Board and bring a wealth of experience to Wipro, having spent 25 years in the global financial services sector. Ms. Naidu is CEO Asia Pacific of Zurich Insurance Group (Zurich) and a member of Zurich’s Executive Committee. She joined Zurich in September 2016 and was named the CEO of the UK business in November 2016 where she implemented an extensive transformation program – reshaping the business, simplifying the structure, improving technical and digital capabilities, and positioning it for growth in its core markets. Before joining Zurich, Ms. Naidu spent 14 years at Prudential in various executiv

REPLY: The Board of Directors Approves the Quarterly Report Dated 31 March 202114.5.2021 12:49:00 CEST | Press release

Today, the Board of Directors of Reply S.p.A. [MTA, STAR: REY] approved the results as at 31 March 2021. Since the beginning of the year, the Group has recorded a consolidated turnover amounting to €352.4 million, an increase of 11.2% compared to the corresponding data for 2020. All indicators are positive for the period. In the first quarter of 2021 the consolidated EBITDA was €59.6 million compared to €47.6 million in 2020, equal to 16.9% of the turnover. EBIT, from January to March, was €47.9 million (€38.0 million in 2020), and is equal to 13.6% of the turnover. The profit before tax, from January to March 2021, was €47.6 million (€33.9 million in 2020), equal to 13.5% of the turnover. The net financial position of the Group on 31 March 2021 is also positive by 227.9 million. The net financial position on 31 December 2020 was positive for €158.7 million. “"The first quarter of 2021 - stated Mario Rizzante, Reply's Chairman - was very positive, both in terms of revenue and margin gr

PMC Biogenix Announces Price Increase on Armoslip® and Kemamide® Fatty Amides14.5.2021 11:00:00 CEST | Press release

PMC Biogenix Inc. announces that effective June 1, 2021 or as contracts permit, prices for Armoslip and Kemamide brand primary and secondary fatty amides and bisamides will increase by $1.00 to $3.00 per kilo for all orders, globally; adjusted for currency fluctuations. This increase is necessary to counterbalance the unprecedented and rapid rate of raw material and logistical cost increases and to continue providing the world class service expected by our global customer base. Customers with questions should contact their local sales manager. About PMC PMC Group is a growth oriented, diversified, global chemicals and plastics company dedicated to innovative solutions to everyday needs in a broad range of end markets including plastics, consumer products, electronics, paints, packaging, personal care, food, automotive and pharmaceuticals. The Company was built on a sustainable model of growth through innovation while promoting social good. PMC operates from a global manufacturing, inno

Mavenir Wins Award for Multi-G Open RAN Small Cell Solutions14.5.2021 09:00:00 CEST | Press release

Mavenir, the Network Software Provider building the future of networks with cloud-native software that runs on any cloud and transforms the way the world connects, is pleased to announce that it has won the Small Cell Forum Judges’ Choice award for its role in transforming mobile networks through its Multi-G Open RAN small cell solutions and its Mobile Network Operator (MNO) Open RAN neutral host integration. Multi-G Open RAN small cell solutions: Adding 2G/3G across a single unified RAN to support Multi-G radio access in 2020, Mavenir extended its Open Radio Access Network (Open RAN) capabilities across 2G, 3G, 4G and 5G to offer MNOs and enterprises a complete end-to-end, software-centric network proposition, spanning small cell radios to converged packet core to digital enablement solutions. This enables the move towards one network, rather than managing cost and complexity of several different networks. With a seamless, multi radio access technology (Multi-RAT) single RAN solution,

Swimlane and Elastic Partner to Deliver an Extensible Framework for Security Operations Teams13.5.2021 22:30:00 CEST | Press release

Swimlane, provider of the industry's leading security automation platform, and Elastic (NYSE: ESTC), the company behind Elasticsearch and the Elastic Stack, today announced a strategic partnership to help global security teams break down silos in their security processes, and provide a force multiplier to security operations teams that are perpetually overwhelmed. This partnership will enhance existing product integrations and jointly develop new capabilities to help security teams be more efficient and effective in protecting their organizations. The combined power of scale and automation Key integrations will enable even highly distributed security operations teams to significantly reduce friction associated with context-gathering tasks and threat containment — providing critical time savings that help analysts triage alerts quickly while minimizing damage from threats. Swimlane and Elastic plan further enhancements to the product experience through: An extensible framework that exte

IFF Achieves Key 2020 Sustainability Goals13.5.2021 22:15:00 CEST | Press release

IFF (NYSE:IFF) today announced the achievement of key 2020 sustainability goals that were set at the launch of the Company’s sustainability program in 2010. During that time, IFF met and surpassed its original intensity-based targets for three environmental goals, resulting in a reduction in its greenhouse gas (GHG) emissions, water withdrawal and hazardous waste generation per metric ton of production by -42.6%, -67.1% and -41% below 2010 levels, respectively. IFF has also achieved its 2020 goal to have 100% of the palm oil used in its products be certified according to the Roundtable on Sustainable Palm Oil (RSPO) Mass Balance Supply Chain Model. These and other milestones are captured in its latest Sustainability Report, “For a Better World,” which is published today. The title references the Company’s purpose – Applying science and creativity for a better world – a clear and bold aspiration that inspires everyone at IFF to push past traditional boundaries and to be a force for a be