Business Wire

Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation, to include children ages 6 through 11. In Europe, there are approximately 3,400 children ages 6 through 11 with two copies of this mutation. Existing reimbursement agreements in countries such as Ireland will enable rapid access to ORKAMBI for these children. In other countries across the European Union, Vertex will now begin the country-by-country reimbursement process.

“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to ORKAMBI,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”

The European Commission’s decision is based on data from two Phase 3 studies of ORKAMBI in children with CF ages 6 through 11 who have two copies of the F508del mutation. In 2017, The Lancet Respiratory published 24-week data from one of these studies, which demonstrated statistically significant improvements in lung function (as assessed by the absolute change in lung clearance index, or LCI2.5, and predicted forced expiratory volume in one second, or ppFEV1) among children treated with ORKAMBI compared to placebo. Improvements in body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score were also observed, although they were not statistically significant. Safety data were similar to those observed in an earlier Phase 3 open-label safety study in children ages 6 through 11. The most common adverse events that occurred more frequently among those receiving ORKAMBI compared to placebo were infective pulmonary exacerbation, productive cough, nasal congestion, oropharyngeal pain, abdominal pain upper, headache, upper respiratory tract infection and sputum increased.

“A principal goal of treating CF is slowing the progressive lung damage caused by this life-shortening genetic disease while improving health in the short term,” said Professor Marcus Mall, M.D., Director of the Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis Center at the Heidelberg University Hospital, Germany. “Studies of ORKAMBI in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.”

About Cystic Fibrosis (CF)

CF is a rare, life-shortening genetic disease affecting approximately 75,000 people across North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell 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 mid-to-late 20s.

About ORKAMBI ®  (lumacaftor/ivacaftor)

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is available as tablets and is typically taken twice per day.

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 and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com.

Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), tezacaftor, VX-440, VX-152, VX-659 and VX-445 were discovered by Vertex as part of this collaboration.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the quotes in the second and fourth paragraphs of this press release and statements regarding the timing of access to ORKAMBI for patients 6 through 11 and the country-by-country reimbursement approval process. While the company believes the forward-looking statements contained in this press release are accurate, 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, risks related to commercializing ORKAMBI for patients 6 through 11 in Europe and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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:
Michael Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach Barber, 617-341-6470
or
Media:
mediainfo@vrtx.com
or
Europe & Australia:
Rebecca Hunt, +44 7718 962 690
or
North America:
Megan Goulart, + 1-617-341-6992

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

G42 Launches OpenAI GPT-OSS Globally on Core42's AI Cloud13.8.2025 19:26:00 EEST | Press release

Core42, a G42 company specializing in sovereign cloud and AI infrastructure, announced the availability of OpenAI’s latest open-weight AI models, including gpt-oss-20B and gpt-oss-120B, on the Core42 AI Cloud platform, instantly accessible through the Core42 Compass API. The deployment enables enterprises, researchers, and developers to run the models on a choice of leading silicon platforms with sovereign, scalable, and high-performance capabilities. Integrated into Compass API with the flexibility to access a wide spectrum of high-performance compute platforms, Core42 delivers industry-leading inference speeds of up to 3,000 tokens per second per user, enabling real-time AI at global scale while aligning workloads with the optimal infrastructure for price-performance and scalability. This deployment delivers tailored performance for low-latency inference workloads and applications, reinforcing Core42’s commitment to secure and optimized global sovereign-enabled AI infrastructure. “Co

Cross-Platform Developers Get Full Control Over Game Distribution With New Features for Xsolla Publishing Suite13.8.2025 19:00:00 EEST | Press release

Xsolla, a global commerce company helping developers launch, grow and monetize their games, announces today a major expansion of its Publishing Suite, a one-stop modular solution built for PC, web, and cross-platform games. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250813620658/en/ (Graphic: Xsolla) As development and marketing costs continue to rise, developers and publishers are seeking new high-margin revenue streams that complement their existing strategies on Steam, Epic, and console. The Publishing Suite is uniquely positioned to help teams enable direct-to-consumer monetization regardless of a game's genre, size, and lifecycle stage. With full control over distribution, storefronts, and player engagement, studios can tailor the experience to any business model or use case. The latest updates to the Xsolla Publishing Suite further enhance its value for free-to-play (F2P) and buy-to-play (B2P) games, enabling faste

LambdaTest Unveils the Fourth Edition of the Testµ ('TestMu') Conference in 202513.8.2025 19:00:00 EEST | Press release

LambdaTest, a leading GenAI-native quality engineering platform, is excited to unveil the 4th edition of its flagship Testµ (‘TestMu’) Conference, taking place virtually from August 19–21, 2025. This year’s event is set to host over 50,000 quality, engineering, and DevOps professionals from more than 120 countries, making it one of the largest online global gatherings dedicated to the future of quality engineering. Testµ (‘TestMu’) Conference 2025 is designed to explore cutting-edge trends, emerging tools, and breakthrough innovations shaping the world of technology. With an esteemed lineup of 100+ global speakers, the conference will feature some of the most influential voices in tech, including Angie Jones, Global Vice President of Developer Relations of Block; Bratin Saha, Chief Product and Technology Officer of DigitalOcean; Dana Lawson, Chief Technology Officer of Netlify; Luis Héctor Chávez, Chief Technology Officer of Replit; Subba Ramaswamy, Managing Director of Accenture; and

Incentive and Recognition Insight on a Global Scale13.8.2025 18:20:00 EEST | Press release

The Incentive Marketing Association (IMA) undertook an extensive year-long global research project to deliver data that is designed to provide actionable incentive and recognition strategy for customer engagement and brand loyalty. The Incentive & Recognition Insights 2025 report synthesizes insights from over 600 executives worldwide, offering comprehensive global benchmarks and trends. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250806639332/en/ IMA Global Market Report Cover “We hoped this study would provide a unified voice to represent the global industry," says Fintan Connolly, Consultant at Engage Loyalty, Research Chair, and past President of IMA Global. “We ran a comprehensive survey to get hard numbers and benchmarks, and we collected open-ended feedback and regional insights so we could capture the stories and context behind the numbers." As part of the global report, nine exclusive reports were developed, prov

Celebrating the Osaka-Kansai Expo 2025 “Top Chef in OSAKA 2025” Featuring Massimo Bottura13.8.2025 18:00:00 EEST | Press release

To commemorate the Osaka-Kansai Expo 2025, the Come to Osaka Campaign Executive Committee—led by Osaka Prefecture, Osaka City, and the Osaka Convention & Tourism Bureau—will present a special culinary event: Top Chef in OSAKA 2025, featuring world-renowned Chef Massimo Bottura. From September 9 to 14, 2025, Chef Bottura will offer exclusive lunch and dinner courses at Restaurant SAKURA, Hotel New Otani Osaka. Located on the 18th floor, the elegant French restaurant offers panoramic views of Osaka Castle and lush surroundings. Chef Bottura’s bespoke menu will showcase the rich agricultural and culinary heritage of Osaka, with most ingredients sourced locally. This initiative supports regional farmers, producers, and chefs, while promoting Osaka’s culinary excellence on a global stage. Massimo Bottura is the chef patron of Osteria Francescana, a three-Michelin-starred and Michelin Green Starred restaurant in Modena, Italy, and a two-time winner of The World’s 50 Best Restaurants. He is a

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
World GlobeA line styled icon from Orion Icon Library.HiddenA line styled icon from Orion Icon Library.Eye