Skip to Content

News & Events

Press Releases

Press Releases

Date Title and Summary Additional Formats
Toggle Summary Two Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment Met Primary Endpoints with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis
  - Study in people who have two copies of the F508del mutation demonstrated a mean absolute improvement in ppFEV 1 of 4.0 percentage points compared to placebo (p
View HTML
Toggle Summary Vertex to Acquire CTP-656 from Concert Pharmaceuticals for the Treatment of Cystic Fibrosis
-Vertex to develop CTP-656 for potential use in future combination regimens aimed at treating the underlying cause of CF- -Concert to receive $160 million in cash with potential for $90 million in future regulatory approval milestone payments- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals
View HTML
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).
View HTML
Toggle Summary Positive Phase 3 Study of ORKAMBI® in Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies of the F508del Mutation Supports a Submission to the European Medicines Agency in the First Half of 2017
- Study met primary endpoint with a statistically significant improvement in absolute change in lung clearance index (LCI 2.5 ) compared to placebo through 24 weeks of treatment - - ORKAMBI was well tolerated with safety data that were similar to data from previous Phase 3 open-label safety study -
View HTML
Toggle Summary Vertex Presents Long-Term Data Demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) Show the Potential to Modify the Progression of CF
- 12 abstracts presented at 30 th Annual North American Cystic Fibrosis Conference highlight data from Vertex's CF program - ORLANDO, Fla. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of long-term data demonstrating that ORKAMBI ®
View HTML
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
View HTML
Toggle Summary U.S. Food and Drug Administration Approves ORKAMBI® (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 through 11 have two copies of the F508del mutation in the U.S. - - Vertex revises ORKAMBI revenue guidance for 2016 - BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S.
View HTML
Toggle Summary Vertex Provides Update on Ongoing Phase 3 Program for VX-661 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis
-Results from Part A of Phase 3 study in people with a mutation that results in minimal cystic fibrosis transmembrane conductance regulator (CFTR) protein function do not support continuation of the study- -Enrollment complete in Phase 3 study in people with two copies of the F508del mutation-
View HTML
Toggle Summary Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mRNA Therapeutics™ for Cystic Fibrosis
-Collaboration to explore use of mRNA Therapeutics to treat the underlying cause of CF by enabling cells to produce functional CFTR proteins in the lungs- - Moderna to receive $40 million upfront, made up of a $20 million cash payment and a $20 million convertible note investment, with potential
View HTML
Toggle Summary Vertex Announces Presentations of Data for KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) at European Cystic Fibrosis Society (ECFS) Conference
-Real-world data presented at ECFS show long-term impact of KALYDECO across multiple measures of disease- -Data from Phase 3 safety study of ORKAMBI in children ages 6-11 presented today at ECFS- BASEL, Switzerland --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
View HTML
Toggle Summary U.S. FDA Accepts for Priority Review Supplemental New Drug Application for the Use of ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6 to 11 who have Two Copies of the F508del Mutation
-Approximately 2,400 children ages 6 to 11 have two copies of the F508del mutation in the U.S. - -Target review date of September 30, 2016 set for the FDA's decision on the application- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced that the U.S.
View HTML
Toggle Summary Vertex Receives Australian Approval 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
-In Australia, approximately 1,000 people with CF ages 12 and older have two copies of the F508del mutation- -ORKAMBI reimbursement process already underway in Australia - LONDON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods
View HTML
Toggle Summary Vertex Awards $1 Million in Grants to 18 Non-profit Organizations to Advance Initiatives for People Living with Cystic Fibrosis
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated  (Nasdaq: VRTX) today announced it has awarded the company's 2016 Cystic Fibrosis (CF) Circle of Care grants , totaling approximately $1 million, to 18 non-profit medical, academic, patient and community organizations.
View HTML
Toggle Summary Vertex Receives Complete Response Letter from U.S. FDA for Use of KALYDECO® (ivacaftor) in People with Cystic Fibrosis Ages 2 and Older with One of 23 Residual Function Mutations
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO ® (ivacaftor) in people with cystic
View HTML
Toggle Summary Health Canada Approves PrORKAMBI® (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation
-Approximately 1,500 people in Canada are ages 12 and older and have two copies of the F508del mutation, the most common genetic form of the disease- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has approved Pr ORKAMBI ®
View HTML
Toggle Summary Vertex Receives EU Approval 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
-In Europe, approximately 12,000 people with CF ages 12 and older have two copies of the F508del mutation- -Positive CHMP Opinion was received in September 2015 - -Country-by-country reimbursement process will now begin- LONDON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
View HTML
Toggle Summary Vertex Receives Two EU Approvals for KALYDECO® (ivacaftor) for People with Cystic Fibrosis
- European Commission approves expanded use of ivacaftor in children with cystic fibrosis ages 2 to 5 who have one of 9 gating mutations; approximately 125 children ages 2 to 5 have one of the approved gating mutations in Europe - - European Commission approves expanded use of ivacaftor in people
View HTML
Toggle Summary Vertex and CRISPR Therapeutics Establish Collaboration to Use CRISPR-Cas9 Gene Editing Technology to Discover and Develop New Treatments for Genetic Diseases
-Gene editing technology to be used to discover treatments to address the mutations and genes known to cause and contribute to cystic fibrosis- -Vertex and CRISPR to utilize gene editing approach to discover treatments for genetic diseases, including sickle cell disease- -Companies establish
View HTML
Toggle Summary Vertex Announces Significant Progress in Its Development Efforts to Treat the Cause of Cystic Fibrosis in the Vast Majority of People with the Disease
-Two next-generation correctors to enter clinical development in November; studies of a triple combination of a next-generation corrector with VX-661/ivacaftor planned for 2016 in people with CF- -Treating the underlying cause of CF with ORKAMBI ® (lumacaftor/ivacaftor) or KALYDECO ® (ivacaftor)
View HTML
Toggle Summary Supplemental New Drug Application for Use of KALYDECO® (ivacaftor) in People with Cystic Fibrosis Ages 2 and Older who have One of 23 Residual Function Mutations Accepted for Priority Review by U.S. FDA
-More than 1,500 people with CF are ages two and older and have one of these 23 residual function mutations in the U.S.- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a
View HTML
Toggle Summary Vertex Receives CHMP Positive Opinions for ORKAMBI™ (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) in the European Union
-CHMP recommends Marketing Authorization for lumacaftor in combination with ivacaftor for people with cystic fibrosis ages 12 and older with two copies of the F508del mutation- -CHMP recommends Marketing Authorization for ivacaftor for children with cystic fibrosis ages 2 to 5 with 9 gating
View HTML
Toggle Summary ADDING MULTIMEDIA FDA Approves ORKAMBI™ (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation
-Approximately 8,500 people in the U.S. are ages 12 and older and have two copies of the F508del mutation, the most common genetic form of the disease- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S.
View HTML
Toggle Summary FDA Approves ORKAMBI™ (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation
-Approximately 8,500 people in the U.S. are ages 12 and older and have two copies of the F508del mutation, the most common genetic form of the disease- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S.
View HTML
Toggle Summary Vertex Announces Data Presentations at European Cystic Fibrosis Society (ECFS) Conference
-Analysis from TRAFFIC and TRANSPORT extension study of lumacaftor in combination with ivacaftor showed that improvements in lung function and other measures of disease were maintained through 48 weeks in people with cystic fibrosis who have two copies of the F508del mutation- BRUSSELS --(BUSINESS
View HTML
Toggle Summary Vertex and Parion Sciences Establish Collaboration to Develop Epithelial Sodium Channel (ENaC) Inhibitors in Cystic Fibrosis and Other Pulmonary Diseases
-ENaC inhibition aims to restore or improve hydration of cell surfaces in the lungs to improve lung function- -Parion to receive $80 million up-front payment with potential for additional development and regulatory milestones and royalty payments- BOSTON & DURHAM, N.C.
View HTML
Toggle Summary New England Journal of Medicine Publishes Data from Two Phase 3 Studies of ORKAMBITM (lumacaftor/ivacaftor) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the  New England Journal of Medicine (NEJM)  published data from the two Phase 3 studies of ORKAMBI TM (lumacaftor/ivacaftor), an investigational medicine designed to treat the underlying cause of
View HTML
Toggle Summary Food and Drug Administration Advisory Panel Voted 12 to 1 to Recommend Approval of ORKAMBI™ (lumacaftor/ivacaftor) to Treat People with Cystic Fibrosis Ages 12 and Older Who Have Two Copies of the F508del Mutation
- FDA decision expected by July 5, 2015 PDUFA date- -Approximately 8,500 people with cystic fibrosis in the U.S. have two copies of the F508del mutation and are ages 12 and older- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S.
View HTML
Toggle Summary Vertex Announces Data from 12-Week Phase 2 Safety Study of VX-661 in Combination with Ivacaftor in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
-The combination was generally well tolerated and all patients completed 12 weeks of treatment- -Mean within-group absolute improvement from baseline in lung function of 4.4 (p=0.009) and 3.0 (p=0.026) percentage points at week 4 and through 12 weeks of treatment, respectively, in patients who
View HTML
Toggle Summary Vertex Receives U.S. Food and Drug Administration Approval of KALYDECO® (ivacaftor) for Children with Cystic Fibrosis Ages 2 to 5 who have Specific Mutations in the CFTR Gene
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved KALYDECO ® for use in children ages 2 to 5 with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance
View HTML
Toggle Summary U.S. Food and Drug Administration Approves KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation
--Approximately 500 people with cystic fibrosis ages 6 and older have the R117H mutation in the United States -- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated  (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved a supplemental new drug
View HTML
Toggle Summary Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
-U.S. submission includes request for Priority Review; Accelerated Assessment has been granted in the EU- -Approximately 8,500 people in the U.S. and 12,000 in Europe ages 12 and older have two copies of the F508del mutation- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:
View HTML
Toggle Summary FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation
BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration's Pulmonary Allergy Drugs Advisory Committee (PADAC) voted 13-2 to recommend approval of KALYDECO ® (ivacaftor) in people with cystic fibrosis (CF) ages 6 and
View HTML
Toggle Summary Vertex Reviews Recent Progress and Announces Upcoming Milestones in the Development of Multiple Combinations of Medicines that Target the Underlying Cause of Cystic Fibrosis
-Data from across Vertex's cystic fibrosis research and development programs to be presented at the 28 th Annual North American Cystic Fibrosis Conference (NACFC) beginning today- -Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements
View HTML
Toggle Summary Vertex Receives European Approval for KALYDECO™ (ivacaftor) in Eight Non-G551D Gating Mutations
-In Europe, approximately 250 people ages 6 and older have one of 8 additional gating mutations- -KALYDECO is the first medicine to treat the underlying cause of CF in people with specific non-G551D gating mutations- EYSINS, Switzerland --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated
View HTML
Toggle Summary Vertex Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration for Use of KALYDECO® (ivacaftor) in People 18 and Older with Cystic Fibrosis who have the R117H Mutation
-Marketing Authorization Application (MAA) variation in Europe planned for third quarter of 2014- BOSTON --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S.
View HTML
Toggle Summary Vertex Receives European CHMP Positive Opinion for KALYDECO™ (ivacaftor) in Eight Non-G551D Gating Mutations
-In Europe, approximately 250 people ages 6 and older have one of 8 additional gating mutations- -KALYDECO is the first medicine to treat the underlying cause of CF in people with non-G551D gating mutations- EYSINS, Switzerland --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
View HTML
Toggle Summary Two 24-Week Phase 3 Studies of Lumacaftor in Combination with Ivacaftor Met Primary Endpoint with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation
-Combination of lumacaftor and ivacaftor is the first regimen designed to treat the underlying cause of CF in people with two copies of the F508del mutation, the most common form of the disease- -All four 24-week treatment arms achieved primary endpoint of mean absolute improvement in FEV 1
View HTML
Toggle Summary Vertex Announces a Signed Letter of Intent to Enable Public Reimbursement of KALYDECO® (ivacaftor) in Canada for Eligible People with Cystic Fibrosis
-Before patients can get access through public reimbursement, each participating province or territory must decide to reimburse KALYDECO through its individual drug program- -Approximately 100 people ages 6 and older in Canada have the G551D mutation - BOSTON --(BUSINESS WIRE)-- Vertex
View HTML
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
View HTML
Toggle Summary Proof-of-Concept Study of Ivacaftor Monotherapy Showed Improvements in Lung Function After Two Weeks of Treatment in People with Cystic Fibrosis who have a Residual Function Mutation
-Data are consistent with in vitro observations in residual function mutations showing that ivacaftor improved CFTR activity- -8-week open-label period showed improvements in lung function- -Data support plans to initiate a Phase 3 study of ivacaftor in people with CF who have a residual function
View HTML
Toggle Summary Addition of VX-661 to KALYDECO® (ivacaftor) Improves Lung Function in People with CF Who Are Heterozygous for the F508del and G551D Mutations in 28-day Phase 2 Proof-of-Concept Study
-Data provide proof-of-concept for use of VX-661 to further enhance CFTR function in people taking KALYDECO who have the F508del mutation and another mutation known to respond to KALYDECO alone- -Statistically significant improvements in lung function, as well as decreases in sweat chloride,
View HTML
Toggle Summary Vertex Reports First Quarter 2014 Financial Results and Provides Updates on Key Business Priorities
-Net product revenues of $100 million for KALYDECO in cystic fibrosis- -Cash, cash equivalents and marketable securities of $1.32 billion on March 31, 2014 - -Six-month dosing period complete for Phase 3 TRAFFIC and TRANSPORT studies of lumacaftor in combination with ivacaftor for people with CF
View HTML
Toggle Summary U.S. Food and Drug Administration Approves KALYDECO™ (ivacaftor) for Use in Eight Additional Mutations that Cause Cystic Fibrosis
-KALYDECO is the first medicine to treat the underlying cause of CF for people with specific mutations in the CFTR gene- -KALYDECO facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein- -The eight additional mutations are present in
View HTML
Toggle Summary Vertex Announces Results of Phase 3 Study of Ivacaftor in People with CF who have the R117H Mutation
-Mean absolute treatment difference in lung function across all patients was 2.1 percentage points (p=0.20) and mean relative treatment difference was 5.0% (p=0.06)(n=69); study did not meet its primary endpoint- -Pre-specified analysis of patients 18 years of age and older (n=50) showed
View HTML
Toggle Summary Vertex Focuses Investment on Future Opportunities in Cystic Fibrosis and Other Key Research and Development Programs and Reduces Workforce Related to INCIVEK
-Changes result from the continued and rapid decline in the number of people being treated with INCIVEK as new hepatitis C medicines near approval- -370 positions, including 175 in Massachusetts , to be eliminated, primarily related to support of INCIVEK- CAMBRIDGE, Mass.
View HTML
Toggle Summary Vertex Announces Recent Progress and Upcoming Milestones in Research and Development Programs for Cystic Fibrosis
-Enrollment complete for TRAFFIC and TRANSPORT Phase 3 studies of lumacaftor (VX-809) in combination with ivacaftor for people with two copies of the F508del mutation (homozygous); data expected in mid-2014- -12-week Phase 2 study of VX-661 in combination with ivacaftor planned for people with two
View HTML
Toggle Summary Vertex Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration for KALYDECO™ (ivacaftor) Monotherapy for People with Non-G551D Gating Mutations
-sNDA also includes long-term safety and efficacy data for KALYDECO from PERSIST open-label rollover study- -Marketing Authorization Application (MAA) variation in Europe planned for October 2013 - CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today
View HTML
Toggle Summary Results from Phase 3 Study of Ivacaftor Monotherapy Showed Statistically Significant Improvements in Lung Function in People with non-G551D Gating Mutations
-Mean absolute treatment difference in lung function (percent predicted FEV 1 ) between treatment with ivacaftor and placebo was 10.7% (p < 0.0001); mean relative treatment difference between treatment with ivacaftor and placebo was 14.2% (p < 0.0001)- -Statistically significant improvements
View HTML
Toggle Summary Vertex Receives Australian Approval for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People with a Specific Genetic Mutation (G551D)
-- Approximately 250 people in Australia have the G551D mutation in the CFTR gene -- CAMBRIDGE, Mass. --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods Administration (TGA) of Australia has approved KALYDECO™ (ivacaftor) for people
View HTML
Toggle Summary Treatment with VX-661 and Ivacaftor in a Phase 2 Study Resulted in Statistically Significant Improvements in Lung Function in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
-Treatment with combination of VX-661 and ivacaftor for 28 days in two highest dose groups resulted in mean relative increases in lung function (percent predicted FEV 1 ) of 9.0% (p=0.01) and 7.5% (p=0.02) versus placebo - -VX-661 was generally well-tolerated alone and in combination with
View HTML