Business Wire

Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) to Treat Patients With Cystic Fibrosis Aged 12 to <24 months With Certain Mutations in the CFTR Gene

Jaa

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 the treatment of people with cystic fibrosis (CF) aged 12 to <24 months who have at least one of the following nine 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 aged 12 to <24 months, who have specific mutations in the CFTR gene.

“Cystic fibrosis is a chronic, progressive disease that is present at birth, with symptoms often occurring in infancy, so early treatment is crucial to deliver 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 marks an important step towards allowing young CF patients to benefit from treatment at an early stage of their disease, and brings us one step closer to our goal of treating all people living with CF.”

The submission was supported by data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF aged 12 to <24 months who have one of 10 mutations in the CFTR gene that demonstrated a safety profile consistent with 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 aged two years and older who have one of the nine 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 aged 18 years and older who have an R117H mutation in the CFTR gene.

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 ARRIVAL Study

The ARRIVAL study is an ongoing Phase 3 open-label safety study of 25 children with CF aged 12 to <24 months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H; patients with the R117H mutation were eligible to enroll in regions where ivacaftor is approved for use in patients 2 through 5 years of age with an R117H mutation). The study demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued due to adverse events. Treatment was interrupted in two patients who had elevated liver enzymes greater than eight times the upper limit of normal, but continued to receive ivacaftor after a dose interruption. The most common adverse events (≥30%) were cough (74%), pyrexia (37%), elevated aspartate aminotransferase (37%), elevated alanine aminotransferase (32%) and runny nose (32%). Four serious adverse events were observed in two patients.

Mean baseline sweat chloride for the children in this study was 104.1 mmol/L (n=14). Following 24 weeks of treatment with ivacaftor, the mean sweat chloride level was 33.8 mmol/L (n=14). In the 10 subjects with paired sweat chloride samples at baseline and week 24, there was a mean absolute change of -73.5 mmol/L. Sweat chloride is used as a tool to diagnose infants with CF, where levels greater than or equal to 60 mmol/L indicate that CF is likely, levels of 30-59 mmol/L indicate CF is possible and levels less than 30 indicate that CF is unlikely. These data were presented at the 41st European Cystic Fibrosis Society (ECFS) Conference in June 2018 and published in The Lancet Respiratory Medicine (Volume 6, No 7, July 2018).

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 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 eight 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 marketing approval from the European Commission 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)

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:
Heather Nichols, +1-617-341-6992
or
Europe & Australia:
Marie von Seyfried, + 44 7933 500887

Tietoja julkaisijasta

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Tilaa tiedotteet sähköpostiisi

Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Grid Parity Facilitates PV Innovations in Efficiency Improvements27.6.2019 10:00:00 EESTTiedote

PV professionals and industry leaders, including Vikram Solar, Jinko, Longi, and GCL-Si, have gathered in Shanghai to exchange market insights at a seminar titled “2H19 PV Market Outlook and Forecast.” This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190627005246/en/ Held by PV InfoLink, a leading market research company, the seminar went over issues revolving solar PV, spanning market and product trends, technology innovations, how tracker matters in subsidy-free PV, and policy changes, providing forecast and solutions for the fast-paced industry. Reviewing the first half of this year, PV demand saw continuous growth worldwide. As shown in China’s customs data, the country exported 22.4 GW of modules between this January and April, representing 90% YoY growth from the same period last year. The U.S. and Europe have both witnessed a sustained growth so far. India, one of world’s largest PV markets, is also expected to see high

SES Successfully Renews EUR 1.2 Billion Credit Facility27.6.2019 09:55:00 EESTTiedote

SES S.A. is pleased to announce the successful renewal of the company’s EUR 1,200,000,000 Committed Revolving Credit Facility. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190626005846/en/ SES Successfully Renews EUR 1.2 Billion Credit Facility (Photo: Business Wire) Following strong support from existing lenders, the facility was considerably oversubscribed having received a high level of early bird commitments from a number of banks. The facility is for general corporate purposes and has been structured as a 5-year, multi-currency revolving credit facility with an option to extend until 2026 (5+1+1). The margin for the new facility is 40 basis points (for a Baa2/BBB rating) above EURIBOR and is 5 basis points inside the pricing of the former syndicated and committed credit facility closed in January 2014. Andrew Browne, Chief Financial Officer of SES, commented: “This financing further strengthens our liquidity profile a

Mass Deployments of IoT Solutions Transforming China, Says GSMA27.6.2019 04:30:00 EESTTiedote

Chinese mobile operators are leading the world in the deployment of transformative Internet of Things (IoT) solutions, the GSMA announced today. By connecting the estimated 960 million1 devices via cellular networks, the country is realising the value of the IoT and transforming society through mass market deployments of smart home, smart industry and smart city solutions. According to GSMA Intelligence, China is the world’s largest IoT market with 64 per cent of the 1.5 billion global cellular connections including the rapidly growing Mobile IoT licensed LPWA technologies2. “Backed by proactive government support, China is now at the forefront in the development and mass deployment of innovative and transformative IoT based solutions based on Mobile IoT technology,” said Alex Sinclair, Chief Technology Officer, GSMA. “These new technologies are being implemented across multiple different vertical sectors fundamentally altering the way we live and work, delivering real-time information

Gemalto Instant Connect Signals New Era in Effortless Mobile Connectivity for Consumer eSIM-equipped Devices  27.6.2019 02:00:00 EESTTiedote

Thales announces the launch of Gemalto Instant Connect, a pioneering connectivity solution to simplify the connection of eSIM-enabled devices on a mobile network. While users are spared the inconvenience of establishing a Wi-Fi or Bluetooth connection to kick-start their mobile experience, OEMs and mobile operators can now provision devices with mobile connectivity at their first use, and not during manufacturing, saving both time and cost. Connected PCs and laptops will be the first devices to integrate this faster and more efficient approach to out-of-the-box connectivity for eSIMs. Going forwards, a wide array of OEMs and mobile operators stand to benefit from the solution, which is fully compliant with the latest GSMA Remote SIM Provisioning specifications. Between 2018 and 2022, an estimated 2.3 billion eSIM-compliant consumer devices will be shipped worldwide 1 . To date, eSIM-equipped devices have had to rely on some form of primary connectivity before they are able to activate

Quanterix Announces Agreement to Acquire UmanDiagnostics, World’s Leading Neurofilament Light (Nf-L) Antibody Supplier26.6.2019 23:00:00 EESTTiedote

Quanterix Corporation (NASDAQ: QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, today announced that it has entered into a definitive agreement to acquire privately held UmanDiagnostics AB for $22.5 million, comprised of $16 million in cash plus $6.5 million in Quanterix common stock. Uman is widely recognized to possess the leading antibodies available today to measure neurofilament light (Nf-L) and has become the provider of choice for biopharmaceutical and diagnostic applications. Uman, based in Umeå, Sweden, supplies Nf-L antibodies and ELISA kits, which are widely recognized by researchers and biopharmaceutical and diagnostics companies world-wide as the premier solution for the detection of Nf-L to advance the development of therapeutics and diagnostics for neurodegenerative conditions. Nf-L has seen a dramatic growth in the last three years since Quanterix developed the first assay using Uman’s antibodies that could relia

Elliott Statement on Bayer26.6.2019 19:44:00 EESTTiedote

Elliott Advisors (UK) Limited (“Elliott”), which advises funds that collectively hold shares of Bayer A.G. (“Bayer” or “the Company”) and economic equivalents with an aggregate value of €1.1 billion, acknowledges Bayer’s announcement to create a special litigation committee of the Supervisory Board (the “Special Committee”), and the retention of Mr. John Beisner of Skadden, Arps, Slate, Meagher & Flom LLP, as legal advisor to the Supervisory Board and Special Committee. Elliott welcomes these steps, and is confident that today’s statement marks a step change in Bayer’s approach to addressing the legal challenges currently facing the Company. Elliott believes that the creation of the Special Committee will provide a new level of oversight and a fresh perspective to a litigation strategy in need of a radical overhaul, and help guide the Company towards a rational, fair and swift settlement. The appointment of Mr. John Beisner not only provides Bayer with relevant litigation expertise, bu

Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.

Tutustu uutishuoneeseemme