ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Inco r porated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an
- 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
-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
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).
- 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 -
- 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 ®
-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
-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.
-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-
-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
-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
-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.
-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
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.
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
-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 ®
-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)
- 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
-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
-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)
-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
-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
-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.
-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.
-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
-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.
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
- 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.
-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
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
--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
-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:
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
-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
-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
-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.
-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)
-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
-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
-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
-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
-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,
-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
-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
-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
-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.
-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
-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
-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
-- 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
