Tezacaftor

Vertex to Initiate Phase 3 Development Program for New Once-Daily Triple Combination Regimen in People With Cystic Fibrosis

Retrieved on: 
Wednesday, July 28, 2021
Biotechnology, Other Health, Health, Pharmaceutical, Clinical trials, Drugs, Orphan drugs, Health sciences, Health, Cystic fibrosis, Breakthrough therapy, Combination drugs, Elexacaftor/tezacaftor/ivacaftor, Organofluorides, Tezacaftor, VX, Ivacaftor, the Phase 3 Program, VX-121/Tezacaftor/VX-561, Cystic Fibrosis, THE PHASE 3 PROGRAM, VX-121/TEZACAFTOR/VX-561, CYSTIC FIBROSIS

Complete data from the Phase 2 clinical study of VX-121/tezacaftor/VX-561 and the VX-561 Phase 2 monotherapy study will be presented at a later date.

Key Points: 
  • Complete data from the Phase 2 clinical study of VX-121/tezacaftor/VX-561 and the VX-561 Phase 2 monotherapy study will be presented at a later date.
  • The primary endpoint in both studies is the absolute change from baseline in ppFEV1, which will be analyzed for non-inferiority to TRIKAFTA.
  • Both studies will also assess absolute change from baseline in ppFEV1 and sweat chloride for superiority to TRIKAFTA.
  • The VX-121/tezacaftor/VX-561 program was granted Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration for the treatment of cystic fibrosis.

Vertex Announces National Reimbursement Agreement in France for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) and SYMKEVI® (tezacaftor/ivacaftor) for Eligible Cystic Fibrosis Patients

Retrieved on: 
Monday, June 28, 2021
Biotechnology, Pharmaceutical, Health, Drugs, Orphan drugs, Health sciences, Cystic fibrosis, Health care, Breakthrough therapy, Combination drugs, Vertex Pharmaceuticals, Ivacaftor, Elexacaftor/tezacaftor/ivacaftor, Tezacaftor, Elexacaftor, Cystic Fibrosis, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination With Ivacaftor, SYMKEVI® (tezacaftor/ivacaftor) in Combination With Ivacaftor, CYSTIC FIBROSIS, KAFTRIO® (IVACAFTOR/TEZACAFTOR/ELEXACAFTOR) IN A COMBINATION WITH IVACAFTOR, SYMKEVI® (TEZACAFTOR/IVACAFTOR) IN COMBINATION WITH IVACAFTOR

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced a national reimbursement agreement with the French Health Authorities for the cystic fibrosis (CF) medicines KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor and SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced a national reimbursement agreement with the French Health Authorities for the cystic fibrosis (CF) medicines KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor and SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor.
  • Both medicines will be available for all eligible patients once the agreement has been published in the French Official Journal.
  • Through this national reimbursement agreement, eligible patients 12 years and older now have access to KAFTRIO and SYMKEVI.
  • The reimbursement agreement enables broad access to KAFTRIO for people with CF ages 12 years and older with one F508del mutation and one minimal function mutation.

Global $20 Billion CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, May 4, 2021
Health, Pharmaceutical, Cystic fibrosis, Medical specialties, Branches of biology, Organ systems, Ivacaftor, Tezacaftor

b'The "Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" report has been added to ResearchAndMarkets.com\'s offering.\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.\nThe report has been prepared in a view to deeply profile the current market trends along with the sales insight on 4 commercially available CFTR Modulators with their, market share and the clinical profile.\nThe advent of CFTR modulators in the management of cystic fibrosis has greatly transformed the overall market.

Key Points: 
  • b'The "Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" report has been added to ResearchAndMarkets.com\'s offering.\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.\nThe report has been prepared in a view to deeply profile the current market trends along with the sales insight on 4 commercially available CFTR Modulators with their, market share and the clinical profile.\nThe advent of CFTR modulators in the management of cystic fibrosis has greatly transformed the overall market.
  • Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies are targeted drugs which have been developed by the researchers to correct the malfunctioning protein made by the CFTR gene.
  • The overall CFTR modulator market will also witness high growth rates and rapid approval of several other modulators which are currently under clinical trials.
  • It is expected that CFTR modulator drugs will emerge as a potential drugs in the management of cystic fibrosis in coming years.\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" Report Highlights:\nGlobal CFTR Modulators Market Opportunity: > US$ 20 Billion\nGlobal CFTR Modulators Market Growth: 42% CAGR (2017 -2020)\nGlobal CFTR Modulators Market Growth In 2020: 55%\n'

Eloxx Pharmaceuticals Adds Additional Treatment Arm to Ongoing Phase 2 Clinical Studies for Cystic Fibrosis

Retrieved on: 
Thursday, April 29, 2021
Cystic fibrosis, Orphan drugs, Drugs, Breakthrough therapy, Health sciences, Chemical compounds, Anilides, Ivacaftor, Tezacaftor, Vertex Pharmaceuticals

b"WALTHAM, Mass., April 29, 2021 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today announced the addition of a new study arm in the ongoing global Phase 2 clinical program for ELX-02 for the treatment of cystic fibrosis (CF) in patients with at least one G542X allele.

Key Points: 
  • b"WALTHAM, Mass., April 29, 2021 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today announced the addition of a new study arm in the ongoing global Phase 2 clinical program for ELX-02 for the treatment of cystic fibrosis (CF) in patients with at least one G542X allele.
  • The Phase 2 clinical program now includes a fifth treatment arm to evaluate safety of ELX-02 in combination with Kalydeco (ivacaftor), an FDA-approved CFTR (CF transmembrane conductance regulator) potentiator for the treatment of cystic fibrosis in patients who have at least one mutation in their CF gene amenable to ivacaftor.
  • The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity.\n\xe2\x80\x9cWe are extremely pleased to include this additional treatment arm in the ongoing Phase 2 clinical trial to further explore the potential of ELX-02 to treat cystic fibrosis,\xe2\x80\x9d said Sumit Aggarwal, President and Chief Executive Officer.
  • ELX-02 is in clinical development focusing on cystic fibrosis.

Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis With At Least One F508del Mutation

Retrieved on: 
Friday, March 26, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Drugs, Orphan drugs, Cystic fibrosis, Health sciences, Breakthrough therapy, Health, Combination drugs, Elexacaftor/tezacaftor/ivacaftor, Ivacaftor, Tezacaftor, Elexacaftor, Vertex, Cystic Fibrosis, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination With Ivacaftor

If the European Commission follows the recommendation, the majority of people with CF in Europe will be eligible for the medicine.

Key Points: 
  • If the European Commission follows the recommendation, the majority of people with CF in Europe will be eligible for the medicine.
  • It showed statistically significant and clinically meaningful improvements in primary and key secondary endpoints, including lung function in patients treated with ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.
  • In Europe, KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently licensed for the treatment of people with CF ages 12 years and older with an F/F or F/MF genotype.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Vertex Receives Australian TGA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat People With Cystic Fibrosis Ages 12 Years and Older Who Have At Least One F508del Mutation

Retrieved on: 
Wednesday, March 24, 2021
Health, Genetics, Other Health, General Health, Clinical trials, Pharmaceutical, Biotechnology, Cystic fibrosis, Branches of biology, Medical specialties, Chemical compounds, Cystic fibrosis transmembrane conductance regulator, Ivacaftor, Tezacaftor, Cystic Fibrosis, TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.

Key Points: 
  • It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.
  • Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body.
  • The approval of any new treatment option for people living with cystic fibrosis is always welcome news.
  • In Australia, TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis in People Ages 12 Years and Older

Retrieved on: 
Friday, August 21, 2020
Health, Other Health, Other Science, Research, Science, Pharmaceutical, Biotechnology, Drugs, Breakthrough therapy, Orphan drugs, Health, Combination drugs, RTT, Elexacaftor/tezacaftor/ivacaftor, Tezacaftor, Ivacaftor, Cystic fibrosis, Elexacaftor, Vertex Pharmaceuticals, Cystic Fibrosis, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With ivacaftor

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide.

Key Points: 
  • 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.
  • KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Positive Phase 3 Study Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older With Cystic Fibrosis Who Have One Copy of the F508del Mutation and One Gating or Residual Function Mutation

Retrieved on: 
Monday, July 20, 2020
Health, FDA, Genetics, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Drugs, Chemical compounds, Orphan drugs, Chemistry, Breakthrough therapy, Ivacaftor, Phenols, Elexacaftor/tezacaftor/ivacaftor, Tezacaftor, Vertex Pharmaceuticals, the 445-104 Study, Cystic Fibrosis, TRIKAFTA®

The regimen was generally well-tolerated, and safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA.

Key Points: 
  • The regimen was generally well-tolerated, and safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA.
  • The study is a post-marketing commitment in the U.S. and the results will be submitted to the U.S. Food and Drug Administration.
  • Full study results will be submitted for presentation at a future medical meeting and/or publication.
  • Following the run-in, patients were randomized to receive TRIKAFTA or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks.

CHMP Grants Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With KALYDECO® (ivacaftor) in People Ages 12 and Older With Cystic Fibrosis With the Most Common Genotypes

Retrieved on: 
Friday, June 26, 2020
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical trials, Chemical compounds, Chemistry, Organic compounds, Breakthrough therapy, Cyclopropanes, Organofluorides, Ivacaftor, Phenols, Tezacaftor, Cystic fibrosis transmembrane conductance regulator, Pulmonology, Pediatrics, Cystic Fibrosis, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With KALYDECO® (ivacaftor)

In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated.

Key Points: 
  • In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated.
  • The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 12 years and older with an F/F or F/MF genotype are unprecedented.
  • Mall, M.D., Head of Department of Pediatric Pulmonology, Immunology and Critical Care Medicine at Charit University Medical Center Berlin.
  • Both the clinical and patient communities are excited that more people with CF will be able to benefit from CFTR modulators.

AllianceRx Walgreens Prime study demonstrates favorable adherence, but high cost for cystic fibrosis patients prescribed modulator medications

Retrieved on: 
Wednesday, November 6, 2019
Chemical compounds, Chemistry, Organic compounds, Ivacaftor, Cystic fibrosis transmembrane conductance regulator, Tezacaftor, Cystic fibrosis, Lumacaftor/ivacaftor, Lumacaftor, Vertex Pharmaceuticals, Eric Sorscher

ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.

Key Points: 
  • ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.
  • Researchers wanted to understand if patients took their medications as prescribed, as well as the financial impact of the medications on patients.
  • Since the introduction of the first CFTR modulator in 2012 and subsequent CFTR modulator approvals, these medications have significantly changed the treatment of CF.
  • Of the 3,482 patients using CFTR modulator therapies, 50.8% of patients were on lumacaftor/ivacaftor followed by tezacaftor/ivacaftor (24.8%) and ivacaftor (24.4%).