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Toggle Summary Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO® in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations
  -Application to add ~200 non-F508del CFTR mutations to the KAFTRIO ® license- -If approved, ~2,800 people with cystic fibrosis in the European Union ages 2 and above could receive a medicine that treats the underlying cause of their disease for the first time- LONDON --(BUSINESS WIRE)--Nov.
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Toggle Summary Vertex Announces Expansion of Reimbursement Agreement With NHS England to Include KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With KALYDECO® (ivacaftor)
-CF patients in England will be among the first in Europe to benefit from access to KAFTRIO ® , if the medicine is approved by the European Commission - LONDON --(BUSINESS WIRE)--Jun. 30, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has expanded its
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Toggle Summary Vertex Announces FDA Approvals of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Use in People With CF With Certain Rare Mutations
- More than 600 people with certain rare CF mutations are now eligible for TRIKAFTA, SYMDEKO or KALYDECO - BOSTON --(BUSINESS WIRE)--Dec. 21, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA ®
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Toggle Summary Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes (T1D)
- Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 - - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D - BOSTON --(BUSINESS WIRE)--Jan. 28, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
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Toggle Summary Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes
  – Vertex to initiate a Phase 1/2 clinical trial in the coming months – – VX-264 is the second investigational program in Vertex’s pipeline containing stem cell-derived, fully differentiated pancreatic islet cells for the treatment of type 1 diabetes – – VX-264 program does not require
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Toggle Summary Vertex Announces FDA Clearance of Investigational New Drug Application for VX-407 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
– ADPKD is the most common inherited kidney disease, with no treatments currently available that address the underlying cause of disease – – Vertex to initiate a Phase 1 clinical trial in healthy volunteers this month – – ADPKD is Vertex’s 10th disease area in the clinic, further expanding the
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Toggle Summary Vertex Announces FDA Fast Track Designation and Initiation of a Phase 1/2 Clinical Trial for VX-880, a Novel Investigational Cell Therapy for the Treatment of Type 1 Diabetes
– VX-880 is the first investigational stem cell-derived therapy utilizing fully differentiated, insulin-producing pancreatic islet cells for the treatment of type 1 diabetes – – VX-880 is the first and only pancreatic islet replacement therapy known to receive Fast Track Designation – BOSTON
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Toggle Summary Vertex Announces FDA Has Lifted the Clinical Hold on VX-880 Phase 1/2 Clinical Trial for the Treatment of Type 1 Diabetes
BOSTON --(BUSINESS WIRE)--Jul. 5, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated
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Toggle Summary Vertex Announces Further Expansion in the Boston Seaport at Dedication of the Jeffrey Leiden Center for Cell and Genetic Therapies
-New 344,000 square foot cell and genetic therapies research and manufacturing facility to be built- -Facility to support continued R&D growth- BOSTON --(BUSINESS WIRE)--May 17, 2022-- At a dedication today of the new Jeffrey Leiden Center for Cell and Genetic Therapies in Boston’s Seaport, Vertex
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Toggle Summary Vertex Announces German Reimbursement Agreement for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation
LONDON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced it has reached a pricing and reimbursement agreement for ORKAMBI ® (lumacaftor/ivacaftor) with the German Federal Association of the Statutory Health Insurances (GKV-SV).
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Toggle Summary Vertex Announces Inaxaplin (VX-147) Granted Breakthrough Therapy Designation by U.S. FDA and Priority Medicines (PRIME) Designation by the EMA
- Vertex granted nine Breakthrough Therapy Designations and three PRIME designations across its pipeline programs to date – BOSTON --(BUSINESS WIRE)--Jun. 8, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has granted inaxaplin
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Toggle Summary Vertex Announces Initiation of Pivotal Phase 3 Program of VX-809 in Combination with Ivacaftor for the Treatment of People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
-Global studies to evaluate two different doses of VX-809 in combination with ivacaftor- - 24-week safety and efficacy data and submission of New Drug Application expected in 2014- CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the initiation
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Toggle Summary Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI® (lumacaftor/ivacaftor) and SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Cystic Fibrosis Patients
Agreement also enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland LONDON --(BUSINESS WIRE)--Apr. 21, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has
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Toggle Summary Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA
- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators - - VX-522 clinical trial in people with CF to initiate in coming weeks - BOSTON --(BUSINESS WIRE)--Dec. 12, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that
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Toggle Summary Vertex Announces it will Submit Cystic Fibrosis Medicines ORKAMBI® (lumacaftor/ivacaftor) as well as SYMKEVI® (tezacaftor/ivacaftor) to be Used in Combination with ivacaftor, to the Scottish Medicines Consortium for Appraisal
- If accepted by the Scottish Medicines Consortium (SMC) for use on the NHS in Scotland , eligible patients could have access to these precision cystic fibrosis medicines in 2019 - - While the SMC reviews the submissions, clinicians can apply for access to these CF medicines via the PACS Tier 2
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Toggle Summary Vertex Announces Key 2012 Business Objectives as Company Prepares for Planned Global Launch of KALYDECO in Cystic Fibrosis
-More than 25,000 people have started treatment for hepatitis C with INCIVEK ® , positioning Vertex for continued growth, earnings and cashflow in 2012- -Preparations for approval and launch of KALYDECO TM ongoing; additional studies of KALYDECO planned for mid-2012- -Nine new medicines in
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Toggle Summary Vertex Announces Key Business Objectives To Support Planned Launch of Telaprevir in Hepatitis C and Continued Progress in Other Serious Diseases
-Hepatitis C: Submission of New Drug Application complete for telaprevir- -Cystic Fibrosis: First Phase 3 data for VX-770 expected in first quarter 2011- -Additional ongoing trials in HCV, CF, epilepsy and rheumatoid arthritis- -Vertex enters 2011 with cash and cash equivalents position of more
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Toggle Summary Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor)
- The agreement allows for reimbursement of ORKAMBI for people with cystic fibrosis who have two copies of the F508del mutation from July 1 - - A framework for assessment and access to our future cystic fibrosis medicines is included as part of the agreement - LONDON --(BUSINESS WIRE)--Jun.
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Toggle Summary Vertex Announces Long-Term Reimbursement Agreement with the Republic of Ireland for ORKAMBI® (lumacaftor/ivacaftor), KALYDECO® (ivacaftor) and Future Cystic Fibrosis Medicines
-Agreement provides access to ORKAMBI for people who have two copies of the F508del mutation and expands access to KALYDECO for all eligible patients- LONDON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced it has reached an agreement with the Health Service
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Toggle Summary Vertex Announces National Reimbursement Agreement in France for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) and SYMKEVI® (tezacaftor/ivacaftor) for Eligible Cystic Fibrosis Patients
- With this reimbursement agreement more than 1,500 patients now have access to a CFTR modulator therapy for the first time - LONDON --(BUSINESS WIRE)--Jun. 28, 2021-- Vertex Pharmaceuticals Incorporated   (Nasdaq: VRTX) today announced a national reimbursement agreement with the French Health
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Toggle Summary Vertex Announces NaV1.8 Inhibitor Advancing to Phase 2 Clinical Development
- Proof-of-concept studies of VX-548 in acute pain to initiate in the second half of the year - BOSTON --(BUSINESS WIRE)--Apr. 26, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it will advance the selective   NaV1.8 inhibitor, VX-548, into Phase 2 clinical
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Toggle Summary Vertex Announces New $750,000 Program to Support the Work of the Next Generation of Cystic Fibrosis Researchers
The Vertex Research Innovation Award (RIA) Program Will Now Provide up to $10M in Grants Over the Next Five Years to New Postdoctoral and Faculty Researchers from Around the World BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated  (Nasdaq: VRTX) announced today a new $750,000 program
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Toggle Summary Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor)
Eligible patients in Scotland will immediately have access to ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor LONDON --(BUSINESS WIRE)--Sep. 12, 2019-- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that eligible cystic fibrosis
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Toggle Summary Vertex Announces New Data on ALS-2200 in People With Hepatitis C, Supporting Advancement into Phase 2 All-Oral Studies in 2012; Ends Development of ALS-2158
- ALS-2158: Well-tolerated in a seven-day viral kinetic study; development discontinued due to lack of efficacy - - ALS-2200: Data from additional cohort of seven-day viral kinetic study with ALS-2200 (200 mg, QD) in combination with ribavirin show median 4.18 log 10 reduction in HCV RNA with 5 of
HCV
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Toggle Summary Vertex Announces New Data that Showed High Viral Cure Rates with a Total of 12 and 24 Weeks of Telaprevir Combination Treatment Among People with Genotype 1 Hepatitis C Who Have the IL28B CC Genotype
- Interim analysis of the Phase 3b CONCISE study showed SVR12 rates of 87 percent with 12 total weeks of treatment and 97 percent with 24 total weeks of treatment among people who achieved RVR and completed 12 weeks of treatment - AMSTERDAM --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated
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Toggle Summary Vertex Announces New Drug Submission for Investigational Triple Combination Medicine for the Treatment of Cystic Fibrosis Has Been Accepted for Priority Review by Health Canada
BOSTON , Dec. 28, 2020 /CNW/ -  Vertex Pharmaceuticals Incorporated   (Nasdaq: VRTX) today announced its New Drug Submission for TRIKAFTA®, Vertex 's investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in
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Toggle Summary Vertex Announces New Portfolio Reimbursement Agreement in Italy Including KAFTRIO®, SYMKEVI® and Additional Indications of ORKAMBI® and KALYDECO® for Eligible Patients With Cystic Fibrosis
- Reimbursement agreement also includes certain future indication extensions across all Vertex CF medicines - - Approximately 1,400 patients will now have access to a CFTR modulator for the first time - LONDON --(BUSINESS WIRE)--Jun. 25, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
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Toggle Summary Vertex Announces Nine Presentations of Data on ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) at the European Cystic Fibrosis Society (ECFS) Conference
- Presentation of data from a Phase 3 study of ORKAMBI in children ages 6-11 with two copies of the F508del mutation demonstrated improvements in lung function and sweat chloride; study also published online in The Lancet Respiratory Medicine today - - ECFS data presentations demonstrate that
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Toggle Summary Vertex Announces Partnership with Basketball Hall-of-Famer Alonzo Mourning to Raise Awareness of APOL1-Mediated Kidney Disease (AMKD)
BOSTON --(BUSINESS WIRE)--Nov. 4, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company has partnered with basketball Hall-of-Famer Alonzo Mourning to launch “Power Forward” ( www.PowerForwardTogether.com ), an educational initiative aimed at raising awareness
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Toggle Summary Vertex Announces Planned Initiation of Phase 2 Studies Evaluating the Next-Generation Correctors VX-440 and VX-152 in Triple Combination Regimens to Treat the Underlying Cause of Cystic Fibrosis
-VX-440 to be evaluated as part of 4-week triple combination dosing with tezacaftor (VX-661) and ivacaftor; VX-152 to be evaluated as part of 2-week triple combination dosing- -Studies to enroll people with cystic fibrosis who have one copy of the F508del mutation and a minimal function mutation
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Toggle Summary Vertex Announces Plans to Enroll Additional Treatment Arm in Ongoing Phase 2 Combination Study of Telaprevir and VX-222 for the Treatment of People with Hepatitis C
-New treatment arm to evaluate all oral, triple combination regimen of telaprevir, VX-222, and ribavirin- CAMBRIDGE, Mass., Nov 10, 2010 (BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced plans to enroll an additional treatment arm as part of its ongoing Phase 2
HCV
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Toggle Summary Vertex Announces Positive 28-Day Results for VX-770, an Oral Investigational Agent That Targets a Defective Protein Responsible for Cystic Fibrosis
<ul><li>VX-770 was well-tolerated when dosed as 150 mg and 250 mg twice daily for 28 days</li><li>VX-770 resulted in a significant increase in FEV1, and significant changes in sweat chloride and nasal potential difference, important markers of CFTR function</li> <li>Researchers to present 28-day results at the North American Cystic Fibrosis Conference on October 23</li></ul>
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Toggle Summary Vertex Announces Positive Day 90 Data for the First Patient in the Phase 1/2 Clinical Trial Dosed With VX-880, a Novel Investigational Stem Cell-Derived Therapy for the Treatment of Type 1 Diabetes
- First patient dosed with VX-880 demonstrated restoration of insulin production and achieved C-peptide of 560 pmol/L in response to Mixed Meal Tolerance Test (MMTT) at Day 90 Visit - - 91% decrease in daily insulin requirement and simultaneous robust improvements in glucose control as measured by
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Toggle Summary Vertex Announces Positive Interim Results with Telaprevir-based Therapy in Genotype 1 Chronic Hepatitis C Patients who Failed to Achieve SVR with Previous Pegylated Interferon & Ribavirin Treatment
-Interim results to be presented in late-breaker poster<br> presentation at EASL on April 24-<br><br> -Significant early on-treatment viral response from patients who<br> previously failed therapy-
HCV
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Toggle Summary Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min)
-Phase 2 data showed mean absolute improvements in ppFEV 1 of 9.7 and 12.0 percentage points for VX-152 and VX-440, respectively, in triple combination with tezacaftor and ivacaftor in F508del/Min patients; Initial data from Phase 1 study showed mean absolute improvement in ppFEV 1 of 9.6
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Toggle Summary Vertex Announces Positive Phase 2 Data in Third Proof-of-Concept Study with the NaV1.8 Inhibitor VX-150
-Treatment with the NaV1.8 inhibitor VX-150 showed significant relief of pain in patients with small fiber neuropathy and was generally well tolerated- -Third positive Phase 2 proof-of-concept study for VX-150 further validates the potential role of NaV1.8 inhibition for the treatment of multiple
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Toggle Summary Vertex Announces Positive Phase 3 Study for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children Ages 6-11 Years With Cystic Fibrosis to Support Submissions for Global Regulatory Approvals
BOSTON --(BUSINESS WIRE)--Sep. 10, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the company has completed a global Phase 3 study of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)   in children ages 6 through 11 years old with cystic fibrosis (CF) who have
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Toggle Summary Vertex Announces Positive Results for VX-770, an Oral Investigational Agent That Targets a Defective Protein Responsible for Cystic Fibrosis
--Interim analysis of 14-day study of VX-770 shows a significant<br> improvement in lung function --<br><br> --Lung function data supported by corresponding improvements in<br> disease-related biomarkers--<br><br> --Results to be shared with health authorities in order to<br> identify the most rapid path forward --
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Toggle Summary Vertex Announces Positive Results from Open-Label Phase 3 Study of KALYDECO® (ivacaftor) in Children with Cystic Fibrosis Ages 1 to 2 Years
-Study met primary safety endpoint and showed improvements across multiple endpoints, including measures of pancreatic function- -Potential to modify the course of CF in children as young as one year of age- -Results support FDA and EMA filings in the first quarter of 2018- BOSTON --(BUSINESS
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Toggle Summary Vertex Announces Positive Results From Phase 2 Study of VX-147 in APOL1-Mediated Focal Segmental Glomerulosclerosis
– Treatment with VX-147 led to a statistically significant, substantial and clinically meaningful mean reduction in proteinuria of 47.6% at 13 weeks compared to baseline and was well tolerated – – Results provide clinical proof of concept for the first genetically targeted approach to treating
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Toggle Summary Vertex Announces Positive Results From Phase 2 Study of VX-548 for the Treatment of Painful Diabetic Peripheral Neuropathy
– Treatment with the NaV1.8 inhibitor VX-548 led to statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the Numeric Pain Rating Scale (NPRS) – – VX-548 was generally well tolerated – – Vertex plans to advance VX-548 into pivotal
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Toggle Summary Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis
– Treatment with the once-daily vanza triple CFTR modulator regimen met all primary and key secondary endpoints in two randomized controlled trials in people with CF ages 12 years and older – – Results were more pronounced in the single-arm study in children ages 6 to 11 years, demonstrating the
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Toggle Summary Vertex Announces Positive Results From the VX-548 Phase 3 Program for the Treatment of Moderate-to-Severe Acute Pain
– Treatment with VX-548 led to statistically significant improvement in pain compared to placebo as well as a clinically meaningful reduction in pain from baseline in both the abdominoplasty and bunionectomy randomized controlled trials – – Treatment with VX-548 was also shown to be effective in
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Toggle Summary Vertex Announces Positive Results from Viral Kinetic Study of the Nucleotide Analogue ALS-2200 in People with Hepatitis C
- 4.54 log 10 median reduction in HCV RNA observed in people with genotype 1 hepatitis C treated with a once-daily 200 mg dose of ALS-2200 for seven days; treatment was well-tolerated- - Phase 2 studies of 12-week all-oral regimens planned for this year - CAMBRIDGE, Mass.
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Toggle Summary Vertex Announces Presentation of Data at North American Cystic Fibrosis Conference
CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 10 abstracts from its cystic fibrosis (CF) research and development program will be presented at the 26th Annual North American Cystic Fibrosis Conference (NACFC) in Orlando, Fla.
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Toggle Summary Vertex Announces Presentation of New Data for Oral JAK3 Inhibitor VX-509 at Annual Meeting of the American College of Rheumatology
-Final data to be presented from Phase 2 study in rheumatoid arthritis- CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that two abstracts related to its investigational oral JAK3 inhibitor, VX-509, will be presented at the 2011 Annual Meeting
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Toggle Summary Vertex Announces Presentation of New Data from Hepatitis C Development Program at AASLD Annual Meeting
CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 11 abstracts from its hepatitis C research and development program will be presented at The Liver Meeting ® , the 63rd Annual Meeting of the American Association for the Study of Liver
HCV
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Toggle Summary Vertex Announces Presentation of New Data on VX-770 and VX-809 at North American Cystic Fibrosis Conference
CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that nine abstracts from its cystic fibrosis (CF) research program, which is aimed at discovering and developing medicines that target the underlying cause of CF, will be presented at the 25th
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Toggle Summary Vertex Announces Presentation of New KALYDECO™ (ivacaftor) Data at European Cystic Fibrosis Society Conference
-New data showed that KALYDECO reduced the loss of lung function by half over 3 years in people with CF who have the G551D mutation compared to similar untreated patients- -New data from rollover study of ivacaftor in people with the R117H mutation support earlier results from Phase 3 study that
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Toggle Summary Vertex Announces Presentations of Data at North American Cystic Fibrosis Conference that Demonstrate Important Progress Toward Goal of Helping All People with CF
- Data from ongoing extension study of ORKAMBI ® (lumacaftor/ivacaftor) in children ages 6-11 and real-world KALYDECO ® (ivacaftor) data demonstrate long-term safety and other benefits of these medicines - - New data from ongoing extension study of tezacaftor/ivacaftor combination demonstrate
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