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

Phase 2 DESTINY-Gastric01 Trial of DS-8201 Versus Chemotherapy Met Primary Endpoint27.1.2020 10:00:00 EETPress release

Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca today announced positive topline results from the pivotal phase 2 DESTINY-Gastric01 trial of DS-8201, a HER2 directed antibody drug conjugate (ADC), in patients with HER2 positive unresectable or metastatic gastric or gastroesophageal junction cancer that had progressed following two or more treatment regimens including trastuzumab and chemotherapy. The trial met its primary endpoint, achieving a statistically significant and clinically meaningful improvement in objective response rate (ORR), as assessed by an independent review committee, in patients treated with DS-8201 versus investigator’s choice of chemotherapy (irinotecan or paclitaxel monotherapy). DS-8201 also showed a statistically significant and clinically meaningful improvement in overall survival (OS), a key secondary endpoint. The safety profile observed for DS-8201 in DESTINY-Gastric01 was consistent with previous clinical trials. These results c

GSMA Recognizes Turkcell and P.I. Works for the Value of AI Based Network Automation27.1.2020 10:00:00 EETPress release

Turkcell and P.I. Works have been recognized by GSMA Future Networks Programme for achieving exceptional efficiency gains in network operations and ensuring superior customer experience. P.I. Works and Turkcell have successfully shown that the AI based, service aware network automation delivered stronger improvements as compared to conventional automation solutions. The details of the deployed solutions and their benefits are provided in a joint white paper authored by GSMA, Turkcell and P.I. Works under GSMA’s Future Networks Programme. To read the white paper please visit: https://www.gsma.com/futurenetworks/wiki/case-study-ai-use-cases-in-service-assurance/ GSMA Future Networks Programme intends to help operators share best practices and discover new opportunities that will enable them to reduce operational and capital expenditures. Given the success of the project, GSMA invited Turkcell and P.I. Works to join the program and provide insights for the AI-based, Service Aware Automate

HighRes Biosolutions and Novo Nordisk partner to transform drug discovery in automated Lab of the Future24.1.2020 17:00:00 EETPress release

HighRes Biosolutions and Novo Nordisk A/S today announced that they will collaborate to design a state-of-the-art robotic platform for high-throughput biologics engineering and characterisation. The integration of a multi-system robotics platform with sophisticated software control architecture will enable a digital transformation and accelerated discovery for Novo Nordisk’s peptide and large molecule therapeutics research. “In order to maintain our leadership within biologics engineering we are investing in a fully automated and digital design platform. This investment is linked to our build-up in AI and advanced analytics and demonstrates Novo Nordisk’s commitment as a world leading biologics research company,” said Lars Fogh Iversen, senior vice president, Global Research Technologies, Novo Nordisk. The platform will comprise several highly automated and integrated cells for production, characterisation and functional analysis of thousands of biologic compounds every month. Its scop

Conquest Invests in 5 Greenfield Onshore Wind Parks Totalizing 43MW in the Flanders Region24.1.2020 15:37:00 EETPress release

Conquest, acting through the Conquest Sustainable Infrastructure fund, announced the transaction was closed after an exclusive agreement had been reached with ENGIE Electrabel. Conquest acquires 51% of a portfolio expected to provide 111 GWh per annum of renewable power representing the annual electricity consumption of about 32,000 Belgian homes from early 2021 onwards. The projects benefit from 15 to 20-year green certificates local remuneration schemes, and thus generate yearly secured revenues. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005221/en/ Credit: Electrabel While the energy and digital transitions continue to drive the infrastructure sector, Conquest keeps building its diversified sustainable real asset portfolio in Western European markets. The success lies in years of collaboration and long-lasting partnerships with fellow industry experts who can notably facilitate access to new growth opportunities

Fluence Expands EMEA Footprint with Horticulture Partners in Papenburg, Germany24.1.2020 14:00:00 EETPress release

Fluence by OSRAM (Fluence), a leading global provider of energy-efficient LED lighting solutions for commercial cannabis and agriculture production, announced today it is expanding its presence in the Emsland region of Germany through partnerships with growers and other horticulture specialists operating out of Papenburg. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005109/en/ Friedrich Schulz’s 5.5 hectare farm—which grows herbs year-round in addition to lettuce, peppers and chives—utilizing VYPR 2p fixtures. (Photo: Business Wire) Given Papenburg’s decades-long history of attracting horticulture experts, Fluence’s innovative lighting solutions for horticulture are a natural fit for the region’s tomato, cucumber, leafy greens and herb growers. Forging connections with Papenburg’s horticultural leaders Elektro Evers, an engineering firm specializing in greenhouse technology, is Fluence’s exclusive systems integrator

HCL Technologies Honors Global Goodwill Champions at the 2020 World Economic Forum in Davos24.1.2020 12:47:00 EETPress release

HCL Technologies (HCL), a leading global technology company, today presented its Wave Maker Awards 2020, commemorating global goodwill champions from around the world. The award ceremony, which was held at the HCL Pavilion in Davos, celebrated organizations and individuals passionately committed to such noble causes as diversity & inclusion, education & community upliftment, environment & sustainability, and technology for change. Special guest at the awards was Australian cricket legend Glenn McGrath, who is also founder of the McGrath Foundation, which provides support for Australian women diagnosed with breast cancer and their families. The award recipients were; Lois Auta, Founder and Executive Director of Cedar Seed Foundation, which supports persons with disabilities to participate in all aspects of life; Chido Govera, Founder and Director of The Future of Hope Foundation (TFoHF), which supports marginal and vulnerable members of society through entrepreneurial and self-developme

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