Business Wire

VERTEX-PHARMA

13.12.2019 19:57:09 CET | Business Wire | Press release

Share
Vertex and Republic of Ireland Expand Long-Term Cystic Fibrosis Medicines Reimbursement Agreement to Include Triple Combination Therapy for All Eligible Patients Ages 12 and Over Once Licensed

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirmed that, following recent collaborative discussions, it has negotiated an agreement with the Health Service Executive (HSE) in the Republic of Ireland to expand the existing long-term cystic fibrosis (CF) reimbursement agreement to include the triple combination regimen (elexacaftor, tezacaftor and ivacaftor), which is under review and pending approval by the European Medicines Agency, for all eligible patients ages 12 and older in line with the potential future licensed indication.

This expands the existing agreement which had already included access to current and future Vertex medicines for people in Ireland with CF of any age who:

  • Have one of the specified mutations as included in the KALYDECO® marketing authorization (G551D, G551S, S549R, G1244E, S1251N, G1349D, S1255P, G178R or S549N ) or
  • Are homozygous for the F508del mutation or
  • Are heterozygous for the F508del mutation and have a specified mutation (P67L , D579G, D1152H, R117C, 711+3A→G, 2789+5G→A, L206W, S945L, 3272-26A→G, R352Q, S977F, 3849+10kbC→T, A455E or R1070W ) and people ages 18 and older and have an R117H mutation.

This innovative long-term agreement also enabled rapid access for people with these mutations if the labels of the existing medicines were expanded to cover additional age groups and if new Vertex medicines were approved for these populations.

Ludovic Fenaux, Senior Vice President, Vertex International, said, “With this expanded agreement, even more patients in the Republic of Ireland will be among the first in Europe to benefit from the triple combination treatment once the medicine is licensed.”

Triple combination treatment (elexacaftor, tezacaftor and ivacaftor) is not currently licensed for use in the European Union. Vertex submitted a Marketing Authorization Application to the European Medicines Agency on October 31, 2019.

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 four approved medicines in the U.S. and three approved medicines in Europe 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 small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

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 10 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 by Mr. Fenaux in the second paragraph of this press release and statements regarding the expansion of the existing long-term cystic fibrosis reimbursement agreement and Vertex’s Marketing Authorization Application submission to the European Medicines Agency. 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 risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied 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, obtaining approval and commercializing elexacaftor, tezacaftor and ivacaftor in Europe, developing additional medicines to treat cystic fibrosis, and other risks listed under Risk Factors in Vertex's annual report and subsequent 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)

Link:

ClickThru

About Business Wire

Business Wire
Business Wire
101 California Street, 20th Floor
CA 94111 San Francisco

http://businesswire.com

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

Agenus Reports 48% Estimated Three-Year Survival With BOT+BAL in Recurrent Ovarian Cancer at IGCS 20263.10.2026 16:50:00 CEST | Press release

Survival estimate remained at 48% between years two and three, extending evidence of durable benefitResults observed in heavily pretreated, difficult-to-treat population that received a median of four prior lines of therapy; nearly three-quarters of patients had platinum-resistant or refractory cancer Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, today announced three-year follow-up results from the ovarian cancer cohort of its 400+ patient Phase 1b C-800-01 trial. The study evaluated botensilimab (BOT), Agenus’ multifunctional, Fc-enhanced anti-CTLA-4 antibody, in combination with balstilimab (BAL), Agenus’ anti-PD-1 antibody. The results, including three-year survival and outcomes by sensitivity to platinum-based chemotherapy and by prior treatment, were presented at the 2026 International Gynecologic Cancer Society (IGCS) Annual Global Meeting in Montréal, Canada. Among patients evaluable for efficacy, three-year overall survival was 48% (n=35), unchanged from

Andersen Global udvider sin juridiske virksomhed i Pakistan med et nyt medlemsfirma2.10.2026 22:36:00 CEST | Pressemeddelelse

Andersen Global styrker sin juridiske tilstedeværelse i Pakistan, idet Samdani & Qureshi, der har været samarbejdspartner siden 2021, skifter navn til Andersen Legal in Pakistan og bliver den seneste medlemsvirksomhed, der slutter sig til organisationen. Andersen Legal in Pakistan har hovedkontor i Islamabad og rådgiver både indenlandske og internationale klienter, herunder virksomheder, investorer, finansielle institutioner, offentlige enheder, udviklingsorganisationer og privatpersoner, om juridiske, kommercielle og regulatoriske forhold. Firmaet arbejder også med voldgift og tvistbilæggelse og rådgiver om komplekse transaktioner, investeringer, finansiering, projekter og tvister i Pakistan og på tværs af landegrænser. "Vores tilgang bygger på stærkt engagement fra ledelsen, klar kommunikation og disciplineret analyse med fokus på at levere rådgivning, som vores klienter kan omsætte til handling," sagde Nudrat Ejaz Piracha, partner hos Andersen Legal in Pakistan. "Ved at blive en del

emnify Appoints Sacha Herrmann as Chief Financial Officer and Christian Friis as Chief Revenue Officer2.10.2026 17:12:00 CEST | Press release

Appointments expand emnify executive team to accelerate global growth emnify, a global leader in IoT connectivity, today announced the appointments of Sacha Herrmann as Chief Financial Officer and Christian Friis as Chief Revenue Officer, effective immediately. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261001902751/en/ emnify Appoints Sacha Herrmann as Chief Financial Officer and Christian Friis as Chief Revenue Officer The appointments follow the recent naming of Kenneth De Spiegeleire as Chief Executive Officer and Frank Stoecker as Executive Chairman of the Board. Together, these leadership changes strengthen emnify financial, operational, and commercial capabilities as the company enters its next phase of international growth. “We have proven momentum. Our ambition is to now turn it into global category leadership,” said Kenneth De Spiegeleire, CEO of emnify. “That requires sharper focus, disciplined execution, and

Tacton Appoints Former IFS Executive Merlin Knott as Chief Commercial Officer to Accelerate Global Growth and Scale Beyond CPQ2.10.2026 15:00:00 CEST | Press release

Enterprise software veteran to lead Tacton’s united commercial organization and partner expansion worldwide. Tacton, a global leader in Configure, Price, Quote (CPQ) software and configuration solutions for manufacturers of complex products, today announced the appointment of Merlin Knott as Chief Commercial Officer (CCO). In this role, Knott will lead Tacton’s global commercial organization, including marketing, sales, and pre-sales, aligning the teams responsible for bringing Tacton’s solutions to market, and driving value to its customers and partners globally. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261002794882/en/ Merlin Knott, Chief Commercial Officer, Tacton Knott joins Tacton with decades of experience building and scaling commercial organizations across the enterprise software industry. Most recently, he held senior leadership roles at IFS, the world’s leading provider of Industrial AI software, where he led

EDW Presents 2026 Data Quality Award to Repeat Winner Domivest2.10.2026 15:00:00 CEST | Press release

European DataWarehouse (EDW) presented its fifth annual Data Quality Award at the 2026 TSI Congress in Berlin. This year, EDW's Best Data Quality – Deal of the Year (2026) award was presented to Domivest B.V. for its transaction Domi 2023-1 B.V., recognising the firm's outstanding commitment to accurate, complete, and consistent reporting. The award is based on EDW's proprietary data quality scoring methodology, which evaluates transactions against thousands of validation rules and checks. Data quality excellence is not a one-off achievement. The fact that the same issuers continue to rank among the top performers year after year demonstrates that superior data quality is the result of sustained commitment, robust processes, and ongoing investment. "Receiving the Data Quality Award for a second time means a great deal to us. Transparent, accurate loan-level data is key to investor trust in our transactions, and we are grateful to European DataWarehouse for recognising the work our trea

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