VRTX Vertex Pharmaceuticals Incorporated
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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CTX001
Beta-thalassemia
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Phase 1/2
Phase 1/2
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Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
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Elexacaftor, tezacaftor and ivacaftor
Children ages 6 to 11 years who have two F508del mutations and one F508del mutation and one minimal function mutation
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PDUFA
PDUFA
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Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
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CTX001
Sickle cell disease
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Phase 1/2
Phase 1/2
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Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
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TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
Children (6-11 years old) with cystic fibrosis (CF) with two copies of F508del mutation or one copy of F508del mutation and one minimal function mutation
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sNDA Filing
sNDA Filing
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Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
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VX-864
Alpha-1 antitrypsin (AAT) deficiency
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Phase 2
Phase 2
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VX-147
Focal segmental glomerulosclerosis (FSGS)
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Phase 2
Phase 2
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Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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VX-814
alpha-1 antitrypsin (AAT) deficiency
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Phase 2
Phase 2
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Phase 2 trial and development to be discontinued due to safety.
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VX-445 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
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Approved
Approved
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FDA Approval announced October 21, 2019.
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Tezacaftor / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
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Approved
Approved
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Label expanded to now include all patients over 6 years old - June 21, 2019.
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Ivacaftor
Cystic Fibrosis (6-12 mths)
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Approved
Approved
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FDA Approval announced April 30, 2019.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 1-2.
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Approved
Approved
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Approval announced August 15, 2018.
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Ivacaftor
Cystic fibrosis (CF) ages 2 to 5.
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Approved
Approved
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FDA Approval announced August 7, 2018.
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ORKAMBI
Cystic fibrosis (CF) ages 6-11 who have F508del mutation
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Approved
Approved
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Approved September 28, 2016.
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VX-661
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in minimal CFTR Function
|
Phase 3
Phase 3
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Phase 3 trial terminated August 2016
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|
Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in a gating mutation
|
Phase 3
Phase 3
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Phase 3 data released October 25, 2017 - primary endpoint not met.
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KALYDECO (ivacaftor)
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
|
Approved
Approved
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Approved March 17, 2015.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
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Approved
Approved
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Approved July 2, 2015.
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KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
|
CRL
CRL
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CRL issued February 5, 2016.
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KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
|
Approved
Approved
|
Approved December 29, 2014.
|
Latest News
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ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 01, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the companies will host an investor webcast on December 9, 2020 at 8:00 a.m. ET to review clinical data presented during the Plenary Scientific Session at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia. The presentation will include speakers from Vertex and CRISPR Therapeutics as well as Haydar Frangoul M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center, and a principal investigator in the CTX001 clinical studies.
The conference call will be webcast live and a link to the webcast can be accessed on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations and on the Vertex website at www.vrtx.com in the "Investors" section. To access the call via phone, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the companies' websites for approximately 30 days.
This meeting is not an official program of the ASH annual meeting.
About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.CRISPR Therapeutics Forward-Looking Statement
This press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about plans to review data presented at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of CTX001 in patients with sickle cell disease and beta thalassemia, as well as the anticipated speakers participating in the investor webcast. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential that preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not support registration or further development; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.CRISPR THERAPEUTICS® word mark and design logo and CTX001™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.
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 multiple 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 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 diseases. 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Vertex 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, statements regarding the expectations and plans to review data presented at the annual ASH meeting and exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia, and the anticipated speakers participating in the investor webcast. 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, the potential for data from a limited number of patients may not to be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, 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 most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)
CRISPR Therapeutics Investor Contact:
Susan Kim, +1 617-307-7503
[email protected]CRISPR Therapeutics Media Contact:
Rachel Eides
WCG on behalf of CRISPR
+1 617-337-4167
[email protected]Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1 617-341-6108
or
Zach Barber, +1 617-341-6470
or
Brenda Eustace, +1 617-341-6187Media:
[email protected]
or
U.S.: +1 617-341-6992
or
Heather Nichols: +1 617-839-3607
or
International: +44 20 3204 5275
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- The combination therapy is a new treatment option for CF patients who are homozygous for F508del -
- The only medicine to treat the underlying cause of CF in this age group with one F508del mutation and one of 14 residual function mutations -
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor), to include the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
"With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. "Today's approval brings us closer to our ultimate goal of providing medicines for all people with CF."
SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) will be immediately available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.
In Europe, SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) is already approved for the treatment of people with CF ages 12 years and older who have either two copies of the F508del mutation in the CFTR gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR gene results in residual CFTR protein activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
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, sinuses, sweat glands, 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 SYMKEVI® in combination with KALYDECO®
Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
For complete product information including dosing guidance, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
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 multiple 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 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 diseases. 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
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, statements made by Dr. Reshma Kewalramani in this press release, statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to SYMKEVI in combination with KALYDECO for eligible patients ages 6-11 years across countries in Europe, and our plans to secure access to SYMKEVI in combination with KALYDECO for additional eligible patients ages 6-11 years in Europe. 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, risks related to commercializing SYMKEVI in combination with KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that it has been notified of an unsolicited "mini-tender" offer dated November 9, 2020, made by TRC Capital Investment Corporation, an Ontario, Canada, corporation to purchase up to 1,000,000 shares of Vertex common stock. TRC Capital's unsolicited "mini-tender" offer price of $210.00 per share is approximately 4.67% below the $220.28 per share closing price of Vertex's common stock on November 6, 2020, the last trading day prior to the commencement of the offer.
Vertex does not endorse TRC Capital's offer and recommends that Vertex shareholders reject the offer and not tender their shares in response to TRC Capital's unsolicited offer. The "mini-tender" offer is at a price below the market price of shares of Vertex's common stock (as of today's date) and is subject to numerous conditions, including TRC Capital's ability to obtain financing necessary to consummate the offer. Vertex is not affiliated in any way with TRC Capital, the offer, or the offer documentation.
TRC Capital has made many similar "mini-tender" offers for the shares of other companies. "Mini-tender" offers are designed to seek less than five percent of a company's outstanding shares, thereby avoiding many disclosure and procedural requirements of the U.S. Securities and Exchange Commission (SEC) because they are below the SEC's threshold to provide such disclosure and procedural protections for investors.
The SEC has cautioned investors about "mini-tender" offers in an investor alert. The SEC noted that these offers "have been increasingly used to catch investors off guard" and that many investors who hear about "mini-tender" offers "surrender their securities without investigating the offer, assuming that the price offered includes the premium usually present in larger, traditional tender offers."
To read more about the risks of "mini-tender" offers, please review the alert on the SEC's website at http://www.sec.gov/investor/pubs/minitend.htm.
Like TRC Capital's other offers, this "mini-tender" puts individual investors at risk because they may sell their shares at a discount without so realizing. Vertex urges shareholders to obtain current stock quotes for their shares of Vertex common stock, review the terms and conditions to the offer, consult with their broker or financial adviser and exercise caution with respect to TRC Capital's "mini-tender" offer. Shareholders who have already tendered should consider the advisability of withdrawing their shares as permitted under TRC Capital's Offer to Purchase documents.
Shareholders who have already tendered their shares may withdraw them at any time prior to the expiration of the offer, and if TRC Capital has not accepted a shareholder's shares for payment pursuant to the offer, that shareholder may withdraw them at any time after December 21, 2020, until TRC Capital accepts the shareholder's shares for payment, in accordance with TRC Capital's offering documents.. According to the offer documents, the offer is currently scheduled to expire at 12:01 a.m., New York City time, on December 9, 2020.
Vertex encourages broker-dealers, as well as other market participants, to review the SEC's letter regarding broker-dealer "mini-tender" offer dissemination and disclosure at https://www.sec.gov/divisions/marketreg/minitenders/sia072401.htm and the NASD Notice to Members regarding guidance to members forwarding "mini-tender" offers to their customers, which can be found at https://www.finra.org/sites/default/files/NoticeDocument/p004221.pdf.
Vertex requests that a copy of this news release be included with all distributions of materials relating to TRC Capital's "mini-tender" offer related to shares of Vertex's common stock.
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 multiple 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 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 diseases. 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
(VRTX-GEN)
View source version on businesswire.com: https://www.businesswire.com/news/home/20201113005658/en/
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Jefferies Virtual London Health Care Conference on Thursday, November 19, 2020 at 9:05 a.m. ET (2:05 p.m. GMT).
The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.
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 multiple 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 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 diseases. 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram. (VRTX-WEB)
View source version on businesswire.com: https://www.businesswire.com/news/home/20201112005822/en/
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- Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe -
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
"Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older. With today's approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "Today's approval is a testament to our commitment to keep going until all people with CF have a treatment option."
The label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.
KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.
KALYDECO® (ivacaftor) is already approved in Europe for people with CF ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.
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, sinuses, sweat glands, 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)
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.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
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 multiple 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 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 diseases. 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
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, statements made by Dr. Reshma Kewalramani in this press release, and statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to KALYDECO for eligible patients four months of age and older across countries in Europe, and our plans to secure access to KALYDECO for additional eligible patients four months of age and older in Europe. 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, risks related to commercializing KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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