Business Wire

Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) to Treat Eligible Infants with Cystic Fibrosis as Early as 6 Months of Age

Share

Vertex Pharmaceuticals (Europe) Limited today announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KALYDECO® (ivacaftor), to include use in infants with cystic fibrosis (CF) ages 6 months to less than 12 months who have one of the following mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

If the European Commission issues a favorable adoption of the EMA CHMP opinion for the extension of indication, ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF in patients ages 6 months to less than 12 months, who have specific mutations in the CFTR gene.

“Cystic fibrosis is present from birth and symptoms frequently manifest in infancy, which is why it is so important to treat the condition as early as possible” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are committed to treating every person with this life-limiting disease and today brings us one step closer to providing a medicine for very young children with cystic fibrosis.”

The submission was supported by data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF ages 6 months to less than 12 months who have certain mutations in the CFTR gene. The study showed a safety profile similar to that observed in previous Phase 3 studies of older children and adults, and improvements in sweat chloride, a key secondary efficacy endpoint.

Ivacaftor is already approved in Europe for the treatment of CF in patients ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. It is also approved for the treatment of CF in patients 18 years and older who have the R117H mutation in the CFTR gene.

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, sinus, sweat gland, 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 KALYDECO® (ivacaftor)
KALYDECO® (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

People with CF who have specific mutations in the CFTR gene are currently benefiting from ivacaftor in countries across North America, Europe and in Australia.

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 three 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 medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha- 1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

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 nine 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, the statements in the second and third paragraphs of the press release. 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, and other risks listed under Risk Factors in Vertex's annual report and 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.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1-617-341-6108
or
Zach Barber, +1-617-341-6470
or
Leah Gibson, +1-857-526-6027

Media: mediainfo@vrtx.com
or
North America:
Sarah D'Souza, +1-617-341-6341
or
Europe & Australia:
Patricia Dessert, +44 7543 237825

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

Isotropic Systems Signs Contract with SES GS to Initiate Multi-Orbit Trials of Next-Gen Multi-beam Antenna Technologies for U.S. Military24.9.2020 09:50:00 EESTPress release

SES Government Solutions (SES GS), a fully-owned affiliate of SES, and Isotropic Systems, a leading developer of transformational broadband terminal technologies, today announced a two-phased antenna evaluation contract with the U.S. Air Force Research Laboratory, working in close collaboration with the U.S. Army Research Engineering Team, for tests of Isotropic Systems’ multi-beam terminal over SES’s O3b Medium Earth Orbit (MEO) constellation to ultimately unleash next-gen connectivity across the battlefield. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200923006010/en/ Isotropic Systems Terminal (Photo: Business Wire) It is the first customer contract between Isotropic Systems and SES Government Solutions and follows on from the significant developmental partnership currently ongoing between the two companies to produce scalable, cost-effective terminals capable of providing government, military, and commercial access to

Altium To Host PCB Industry's Premier Virtual Event24.9.2020 09:30:00 EESTPress release

AltiumLive, Altium’s annual flagship printed circuit board (PCB) design conference, is transitioning from a live, three-day event into a virtual summit. The free event is open to all, and meticulously curated to preserve the quality and content the electronic design community has come to expect. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200923006009/en/ AltiumLive will provide a dynamic approach to learn, network, and collaborate - from the convenience of either your home or workplace. (Graphic: Altium LLC) “We’re very excited to deliver our unique AltiumLive experience for the first time ever in a virtual format. As always we will provide a dynamic approach to learn, network and collaborate - but this time, from the convenience of either your home or workplace,” said Ted Pawela, Altium’s COO. “By making our conference both virtual and free of charge, it will remove the limitations of time, travel, workload, and budget,

Vifor Pharma announces outcome of AFFIRM-AHF topline data24.9.2020 08:00:00 EESTPress release

Regulatory News: Vifor Pharma today announced topline data from its AFFIRM-AHF study evaluating the effect of Ferinject ® (intravenous ferric carboxymaltose) on heart failure hospitalizations and cardiovascular mortality in iron-deficient patients hospitalized for acute heart failure (AHF), compared to placebo. The trial narrowly missed statistical significance on its composite primary endpoint of reducing the risk of total heart failure hospitalizations and cardiovascular death. A pre-specified sensitivity analysis considering the impact of the COVID-19 pandemic, revealed a statistically significant difference in favor of Ferinject® on cardiovascular mortality and hospitalization for heart failure. The study results will be presented at the American Heart Association (AHA) congress in November this year. “This trial makes a significant contribution to the growing body of evidence showing the importance of detecting and managing iron deficiency in heart failure”, said Prof Piotr Poniko

Taulia’s Darcy Douglas Named One of the Top Women in Supply Chain23.9.2020 20:30:00 EESTPress release

Taulia, the leader in working capital technology solutions, has today announced Darcy Douglas, VP of Professional Services, has been listed as a recipient of Supply & Demand Chain Executive’s first Women in Supply Chain award. The award recognizes female leaders in the supply chain industry who have demonstrated a commitment to excellence in their respective roles and also supporting other women to develop their careers and skills. Darcy has been at Taulia for ten years and her leadership has helped the company grow from a small start-up to an established market leader in the supply chain finance industry. During her time at Taulia, Darcy has spearheaded the creation of three leadership and professional development programs that help Taulians boost their skills and enhance their careers. Specifically, Darcy created the Leadership Initiative for Taulia (LIFT) program for women, which includes a mentoring program, education classes, and other activities, to inspire development and leader

DC Secretary Announces Proposed Amendments to DC Rules23.9.2020 18:22:00 EESTPress release

DC Administration Services, Inc., in its capacity as DC Secretary, has today published to the DC website proposed amendments to the Deliverable Obligation and External Review provisions of the DC Rules. Any Eligible Market Participant may provide comments on the proposed amendments to the DC Rules until 5:00pm EDT Tuesday, September 29, 2020. The proposed amendments to the DC Rules, together with additional information about the Determinations Committees and what they do can be found at the Determinations Committees website: https://www.cdsdeterminationscommittees.org/. Other inquires may be directed to Mark Ferraris at mark.ferraris@citadelspv.com View source version on businesswire.com: https://www.businesswire.com/news/home/20200923005724/en/ Contact information Mark Ferraris mark.ferraris@citadelspv.com

MIRIS: Green Growth in the Midst of a Pandemic23.9.2020 17:19:00 EESTPress release

In March 2020, also Norway ran out of infection control equipment. In this critical phase, MIRIS, a top tier technology green real estate company, initiated a collaboration with China Development Integration Limited (CDIL), a major EPC integration corporation in the Silk Road initiative. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200923005669/en/ From left to right: Henrik Berg Klemmetsen, Magdalena Urbanik, Danny Wong, Andy Lai , Davis Chiu Xinwen Chang and Jan Gunnar Mathisen. Frame: Edmund Amir. (Photo: Business Wire) The two companies have a relationship with regards to building a large-scale smart city in Europe, and MIRIS realized the possibility of a cooperation with CDIL and its resources to ensure supply of high quality infection control equipment. Together they fulfilled significant contracts regarding infection control equipment for several countries, including Norway. In a market described as the "Wild West",

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