Cystic fibrosis transmembrane conductance regulator

Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) in Infants With Cystic Fibrosis Ages 6 Months to Less Than 12 Months With Certain Mutations in the CFTR Gene

Retrieved on: 
Tuesday, December 10, 2019
Biotechnology, General Health, Pharmaceutical, Health, Branches of biology, Chemistry, Organic chemistry, Cystic fibrosis transmembrane conductance regulator, Ivacaftor, Cystic fibrosis, Vertex Pharmaceuticals, Cystic Fibrosis, KALYDECO® (ivacaftor), Vertex

It is also approved for the treatment of CF in patients 18 years and older who have the R117H mutation in the CFTR gene.

Key Points: 
  • It is also approved for the treatment of CF in patients 18 years and older who have the R117H mutation in the CFTR gene.
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.
  • Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines

Retrieved on: 
Wednesday, November 13, 2019
Biotechnology, Pharmaceutical, Health, Cystic fibrosis, Health, Vertex Pharmaceuticals, Life sciences, RTT, VRTX, Cystic fibrosis transmembrane conductance regulator, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that NHS Wales has accepted an offer for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that NHS Wales has accepted an offer for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.
  • Todays announcement is good news for the approximately 270 eligible cystic fibrosis patients in Wales who will soon have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International.
  • We thank the authorities in Wales for their collaboration in accepting this offer under the same terms as were recently announced in England.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

 Vertex Confirms Northern Ireland Offer Accepted for Cystic Fibrosis Medicines

Retrieved on: 
Tuesday, November 12, 2019
Biotechnology, Pharmaceutical, Health, Vertex Pharmaceuticals, Branches of biology, Medical specialties, Life sciences, RTT, Cystic fibrosis transmembrane conductance regulator, VRTX, Cystic fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that the Regional Pharmaceutical Procurement Service in Northern Ireland has accepted an offer for access to all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that the Regional Pharmaceutical Procurement Service in Northern Ireland has accepted an offer for access to all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.
  • We are pleased that the nearly 280 eligible cystic fibrosis patients in Northern Ireland will soon have access to CFTR modulators to treat the underlying cause of their disease and we thank the authorities in Northern Ireland for their collaboration and commitment in this agreement, said Ludovic Fenaux, Senior Vice President, Vertex International.
  • Nearly 480 people in Northern Ireland have CF.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

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%).

Paragon Genomics Introduces CleanPlex® CFTR Panel And Unveils New Fusion Detection Methods At AMP 2019 Annual Meeting

Retrieved on: 
Monday, November 4, 2019
Branches of biology, Biology, Natural sciences, Molecular biology, Laboratory techniques, Biotechnology, DNA, Cystic fibrosis transmembrane conductance regulator, Polymerase chain reaction, Cystic fibrosis, Fusion gene, DNA sequencing

HAYWARD, Calif., Nov. 4, 2019 /PRNewswire/ --Paragon Genomics, Inc., a leader in amplicon-based target enrichment solutions for next-generation sequencing (NGS), today announced the upcoming introduction of two new products at the 2019 Association for Molecular Pathology (AMP) Annual Meeting & Expoa CleanPlex CFTR Panel for cystic fibrosis and CleanPlex Fusion Detection Methods for the diagnostic and prognostic assessment of cancer tumor progression.

Key Points: 
  • HAYWARD, Calif., Nov. 4, 2019 /PRNewswire/ --Paragon Genomics, Inc., a leader in amplicon-based target enrichment solutions for next-generation sequencing (NGS), today announced the upcoming introduction of two new products at the 2019 Association for Molecular Pathology (AMP) Annual Meeting & Expoa CleanPlex CFTR Panel for cystic fibrosis and CleanPlex Fusion Detection Methods for the diagnostic and prognostic assessment of cancer tumor progression.
  • The CleanPlex CFTR Panel covers all exons of the CFTR gene and includes the important American College of Medical Genetics (ACMG)-recommended mutations, in a rapid and easy-to-use format.
  • Paragon Genomics also announced early access to its CleanPlex Fusion Detection Methods for known and novelgene fusions.
  • The CleanPlex Fusion Detection Methods are built upon Paragon Genomics' key ultra-high multiplex PCR and PCR background cleaning technologies, enabling the detection of fusion genes with or without known breakpoints in a rapid and easy-to-use format.

Paragon Genomics Introduces CleanPlex® Cftr Panel And Unveils New Fusion Detection Methods At AMP 2019 Annual Meeting

Retrieved on: 
Monday, November 4, 2019
Branches of biology, Biology, Natural sciences, Molecular biology, Laboratory techniques, Biotechnology, DNA, Cystic fibrosis transmembrane conductance regulator, Polymerase chain reaction, Cystic fibrosis, Fusion gene, DNA sequencing

HAYWARD, Calif., Nov. 4, 2019 /PRNewswire/ --Paragon Genomics, Inc., a leader in amplicon-based target enrichment solutions for next-generation sequencing (NGS), today announced the upcoming introduction of two new products at the 2019 Association for Molecular Pathology (AMP) Annual Meeting & Expoa CleanPlex CFTR Panel for cystic fibrosis and CleanPlex Fusion Detection Methods for the diagnostic and prognostic assessment of cancer tumor progression.

Key Points: 
  • HAYWARD, Calif., Nov. 4, 2019 /PRNewswire/ --Paragon Genomics, Inc., a leader in amplicon-based target enrichment solutions for next-generation sequencing (NGS), today announced the upcoming introduction of two new products at the 2019 Association for Molecular Pathology (AMP) Annual Meeting & Expoa CleanPlex CFTR Panel for cystic fibrosis and CleanPlex Fusion Detection Methods for the diagnostic and prognostic assessment of cancer tumor progression.
  • The CleanPlex CFTR Panel covers all exons of the CFTR gene and includes the important American College of Medical Genetics (ACMG)-recommended mutations, in a rapid and easy-to-use format.
  • Paragon Genomics also announced early access to its CleanPlex Fusion Detection Methods for known and novelgene fusions.
  • The CleanPlex Fusion Detection Methods are built upon Paragon Genomics' key ultra-high multiplex PCR and PCR background cleaning technologies, enabling the detection of fusion genes with or without known breakpoints in a rapid and easy-to-use format.

AbbVie Announces Collaboration with Cystic Fibrosis Foundation

Retrieved on: 
Thursday, October 24, 2019
Cystic fibrosis, Branches of biology, Medical specialties, Health, Cystic Fibrosis Foundation, Cystic fibrosis transmembrane conductance regulator, AbbVie Inc., Vertex Pharmaceuticals, Cystic Fibrosis Canada

NORTH CHICAGO, Ill., Oct. 24, 2019 /PRNewswire/ -- AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, and the Cystic Fibrosis Foundation, announced today a strategic collaboration in which AbbVie will develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Foundation.

Key Points: 
  • NORTH CHICAGO, Ill., Oct. 24, 2019 /PRNewswire/ -- AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, and the Cystic Fibrosis Foundation, announced today a strategic collaboration in which AbbVie will develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Foundation.
  • Under the terms of the agreement, AbbVie will advance the potentiator into clinical development for potential use in combination treatments for cystic fibrosis (CF).
  • "Modulator therapies represent the most transformative treatment advance in the history of CF," said William Skach, M.D., Senior Vice President of Research Affairs, Cystic Fibrosis Foundation.
  • The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis.

Vertex Announces Agreement with NHS England for Access to All Licensed Cystic Fibrosis Medicines

Retrieved on: 
Thursday, October 24, 2019
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Cystic fibrosis, Vertex Pharmaceuticals, Life sciences, Medical specialties, Branches of biology, RTT, Cystic fibrosis transmembrane conductance regulator, VRTX, Ivacaftor, Ataluren

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced an access agreement with NHS England for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced an access agreement with NHS England for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines.
  • Today is a significant day for the cystic fibrosis community in England.
  • This important agreement, reached in collaboration and partnership with NHS England and NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

Cystic Fibrosis Foundation Statement on FDA Approval of TRIKAFTA, the First Triple-Combination Therapy for the Most Common CF Mutation

Retrieved on: 
Monday, October 21, 2019
FDA, Genetics, Philanthropy, Biotechnology, General Health, Pharmaceutical, Health, Foundation, Science, Other Science, Chemical compounds, Chemistry, Organic chemistry, Cystic fibrosis transmembrane conductance regulator, Ivacaftor, Cystic Fibrosis Foundation, Tezacaftor, Cystic fibrosis, Vertex Pharmaceuticals

Todays FDA approval of TRIKAFTA (elexacaftor/ivacaftor/tezacaftor), the highly effective and highly anticipated triple-combination CFTR modulator, marks the start of a new era in cystic fibrosis.

Key Points: 
  • Todays FDA approval of TRIKAFTA (elexacaftor/ivacaftor/tezacaftor), the highly effective and highly anticipated triple-combination CFTR modulator, marks the start of a new era in cystic fibrosis.
  • We at the CF Foundation, and many in the CF community, have dreamed about this milestone for decades, said Dr. Preston Campbell, president and CEO of the Cystic Fibrosis Foundation.
  • We will not rest until every person with CF has a treatment for the underlying cause of their disease and, one day, a cure.
  • TRIKAFTA is approved for people 12 years and older with CF who have at least one F508del mutation in the CFTR gene.

Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor)

Retrieved on: 
Monday, October 21, 2019
Biotechnology, Pharmaceutical, Genetics, Health, Cystic fibrosis, Branches of biology, Cystic fibrosis transmembrane conductance regulator, Proteins, Medical specialties, Pancreas disorders

We thank the Spanish Authorities for their collaboration and commitment to working with us in an innovative way to come to a solution for patients.

Key Points: 
  • We thank the Spanish Authorities for their collaboration and commitment to working with us in an innovative way to come to a solution for patients.
  • We would also like to acknowledge the medical community for their important input during this process.
  • 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.