Business Wire

Vertex Receives European CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease

Share

Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ORKAMBI® (lumacaftor/ivacaftor) for the treatment of people with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease.

If the European Commission issues a favorable adoption of the EMA CHMP opinion for the extension of indication, lumacaftor/ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF for patients aged 2 to 5 years old who have two copies of the F508del mutation.

“Cystic fibrosis is a chronic, progressive disease and it is important to treat early to ensure the best possible outcomes for patients,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Today’s announcement brings us one step closer to providing more young children with a treatment that addresses the underlying cause of their disease. It also marks another important milestone on our path towards our goal of treating every person with this rare and life-limiting disease.”

The submission was supported by data from a Phase 3 open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile in these pediatric patients generally consistent with that in patients aged 6 years and older.

Lumacaftor/ivacaftor is already approved in Europe for the treatment of CF in patients aged 6 and older who have two copies of the F508del mutation.

About CF
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in 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 proteins 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 build-up 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 the Phase 3 open-label safety study
This CHMP positive opinion is based on a Phase 3 open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile similar to that in patients aged 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24 (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6, n=49). Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study. The most common adverse event (≥30% overall) was cough (63%); most adverse events were mild or moderate in severity. Four patients experienced serious adverse events (2 infective pulmonary exacerbations of cystic fibrosis, 1 gastroenteritis viral, 1 constipation) and three patients discontinued treatment due to elevated transaminases without concurrent elevations in total bilirubin. These findings were presented at the 41st European Cystic Fibrosis Society Conference in June 2018.

About ORKAMBI ® (lumacaftor/ivacaftor)
Lumacaftor/ivacaftor 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.

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, Australia and Latin America. 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 nine years in a row.

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, Dr. Kewalramani’s statement in the third paragraph of this press release. While Vertex 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, that Vertex could experience unforeseen delays in obtaining the European Commission favorable adoption of the EMA CHMP opinion for the extension of indication and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. 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, +1-617-341-6108
or
Eric Rojas, +1-617-961-7205
or
Zach Barber, +1-617-341-6470 or

Media: mediainfo@vrtx.com
or
North America:
Sarah D'Souza, + 1-857-329-8495
or
Europe & Australia:
Marie von Seyfried, + 44 7933 500887

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

NetApp Empowers Customers to Securely “Talk to Their Data” in Collaboration with NVIDIA18.3.2024 23:00:00 CET | Press release

NetApp® (NASDAQ: NTAP) today announced it is collaborating with NVIDIA to advance retrieval-augmented generation (RAG) for generative AI applications. The new collaboration directly connects the just-announced NVIDIA NeMo Retriever microservices—coming to the NVIDIA AI Enterprise software platform for development and deployment of production-grade AI applications, including generative AI—to exabytes of data on NetApp’s intelligent data infrastructure. Every NetApp ONTAP® customer will now be able to seamlessly “talk to their data” to access proprietary business insights without having to compromise the security or privacy of their data. Enterprises want to leverage publicly available large language models to talk directly and securely to their corporate data with assurance that their private data will never be accessible outside the enterprise. Until now, enterprises that wanted to build internal chatbots, co-pilots, and applications that leverage corporate knowledge were faced with co

KPMG’s "Digital Gateway for Tax" Platform Raises Global Standards in Tax Functionality with Generative AI18.3.2024 18:38:00 CET | Press release

In a landmark move, KPMG has enhanced the capabilities of its Digital Gateway for Tax incorporating generative AI (genAI) as an independent component to communicate directly with data, enabling powerful insights and enhanced levels of efficiency. This pioneering Software as a Service (SaaS) is designed to provide an end-to-end solution for all members of the tax department and marks a significant milestone in tax technology, integrating generative AI for organizations embarking on their tax digital transformation journey. The KPMG Digital Gateway for Tax platform’s real strength lies in its content and transparent data enrichment capabilities. The solution empowers clients to use the Digital Gateway as their Centralized Knowledge Hub, where users can access company-specific documents through the unified genAI platform. The RAG approach (Retrieval Augmented Generation) is relied upon to effortlessly enrich models with additional content which helps ensure the information used to generat

Media invitation: World leaders must make headway for adaptation - there are no alternatives18.3.2024 18:06:00 CET | Press release

A coalition of international climate organizations will come together for an event in Copenhagen on the 20 March 2024 to deliver recommendations on integrating climate and development for climate finance to world leaders, ahead of a year of intense negotiations, starting at the Copenhagen Climate Ministerial. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240318208761/en/ Photo: Mathilde Utzon The year 2024 is of paramount importance for our future. We face an urgent need to handle the climate crisis, within the broader range of Sustainable Development Goals. This year’s high-level meetings leading up to COP29 will focus on climate finance, aiming to address the substantial funding gaps. To kick-start these discussions DanChurchAid, E3G, the International Institute for Environment and Development, CONCITO, NDC Partnerships, and the UN Foundation are coming together to co-host the virtual and in-person event ‘Integrating Clim

Verimatrix Releases Counterspy as a New Anti-Piracy Solution with First-Ever Defensive Capabilities18.3.2024 17:45:00 CET | Press release

Regulatory News: Verimatrix, (Euronext Paris: VMX), the leader in powering the modern connected world with people-centered security, today announced general availability of its new Verimatrix Counterspy anti-piracy solution -- the most advanced anti-piracy technology seen in the past decade. Counterspy leverages technology first developed by the company's cybersecurity team back in 2021 to offer an innovative new way to counter the rise in video piracy in an era where streaming apps are prevalent. Counterspy is a standalone product within Verimatrix's anti-piracy team, engineered to prevent video content theft, shield apps from attack, and provide ongoing piracy threat prediction, detection and response services. Counterspy goes beyond traditional methods to ensure top-tier security for media app subscribers, safeguarding content across various devices. It fills the gap in authentication created by the shift from operator-controlled set top box hardware to retail or app-based OTT clien

Centrico Spa (Gruppo Banca Sella) and Veracode Enter Deal to Help Secure the Application Development Life Cycle18.3.2024 15:42:00 CET | Press release

Centrico Spa, part of Gruppo Sella and a specialist in providing open banking systems for innovation-oriented financial institutions, has entered a collaboration with Veracode, a global leader in application security risk management. The deal will enhance the analysis, detection, and prioritization of flaws to expedite software fixes at every stage of Centrico’s application development life cycle. Modern banking and finance are exposed to a stringent and evolving landscape, not least as a result of the Digital Operational Resilience Act (DORA) and an increase in frequent and sophisticated cyberattacks. According to data collected in Veracode’s State of Software Security Report, 72 percent of financial organizations contain vulnerabilities—the lowest percentage of all sectors analyzed and an improvement from the previous year. Even so, financial organizations would benefit from increased automation and secure coding techniques to help them prevent, detect, and respond to vulnerabilities

HiddenA line styled icon from Orion Icon Library.Eye