Cystic fibrosis

Arrowhead Pharmaceuticals Presents Preclinical Data on ARO-ENaC at the North American Cystic Fibrosis Conference

Retrieved on: 
Thursday, October 31, 2019
Research, Infectious diseases, Genetics, Clinical trials, Cardiology, Biotechnology, Pharmaceutical, Health, Science, Oncology, Sodium channels, Branches of biology, Membrane proteins, Medical specialties, Electrophysiology, Epithelial sodium channel, Arrowhead Pharmaceuticals, Cystic fibrosis, ENAC

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today presented preclinical data at the 2019 North American Cystic Fibrosis Conference (NACFC) on ARO-ENaC, an inhaled RNAi therapeutic being developed as a potential treatment for cystic fibrosis (CF), which is a rare disease caused by genetic mutations that lead to mucus buildup in the lungs and pancreas.

Key Points: 
  • Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today presented preclinical data at the 2019 North American Cystic Fibrosis Conference (NACFC) on ARO-ENaC, an inhaled RNAi therapeutic being developed as a potential treatment for cystic fibrosis (CF), which is a rare disease caused by genetic mutations that lead to mucus buildup in the lungs and pancreas.
  • Arrowhead is currently conducting IND/CTA-enabling studies to support regulatory filings in the first half of 2020 for first-in-human studies.
  • ARO-ENaC is designed to reduce production of the epithelial sodium channel alpha subunit (ENaC) in the airways of the lung.
  • Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.

Phase 3 Results from Two Studies of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New England Journal of Medicine and The Lancet

Retrieved on: 
Thursday, October 31, 2019
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Organ systems, Medical specialties, Digestive system, RTT, Abdomen, Pharmacology, Endocrine system, Gastrointestinal tract, Acetylcholine, Cystic fibrosis, Cystic Fibrosis, SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), Vertex

CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

Key Points: 
  • CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
  • It is not known if TRIKAFTA is safe and effective in children under 12 years of age.
  • TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works.
  • Patients should not drive a car, operate machinery, or do anything that requires alertness until they know how TRIKAFTA affects them.

Laurent Pharmaceuticals Receives an Additional up to $3M Award from Cystic Fibrosis Foundation

Retrieved on: 
Thursday, October 31, 2019
Biotechnology, Health, Radiology, Pharmaceutical, Clinical trials, Medical specialties, Cystic Fibrosis Foundation, Cystic fibrosis, Health, Organ systems, Cystic Fibrosis Trust, Vertex Pharmaceuticals, Cystic Fibrosis, LAU-7b, Laurent Pharmaceuticals

Laurent Pharmaceuticals Inc. (the Company), today announced that it has reached an agreement to receive an additional funding of up to US$ 3 million from the US-based Cystic Fibrosis Foundation (the Foundation) to further support its APPLAUD Phase 2 clinical study evaluating the Companys lead compound LAU-7b in adult patients with cystic fibrosis (CF).

Key Points: 
  • Laurent Pharmaceuticals Inc. (the Company), today announced that it has reached an agreement to receive an additional funding of up to US$ 3 million from the US-based Cystic Fibrosis Foundation (the Foundation) to further support its APPLAUD Phase 2 clinical study evaluating the Companys lead compound LAU-7b in adult patients with cystic fibrosis (CF).
  • This adds to the US$ 5 million development award already made available by the Foundation.
  • We are grateful for this additional contribution from the Foundation and the continued support received from the Foundations Therapeutic Development Network throughout the course of the ongoing APPLAUD trial, said Radu Pislariu, MD, President and CEO of Laurent Pharmaceuticals.
  • Cystic Fibrosis (CF) is a progressive, life-threatening, genetic disease affecting about 75,000 people worldwide.

Vertex Announces European Medicines Agency Marketing Authorization Application Validation for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Triple Combination Treatment in Cystic Fibrosis

Retrieved on: 
Thursday, October 31, 2019
General Health, Infectious diseases, Other Health, Health, Pharmaceutical, Chemical compounds, Chemistry, Health, Cystic fibrosis, Breakthrough therapy, Vertex Pharmaceuticals, Ivacaftor, European Medicines Agency, Vertex, Tezacaftor, Marketing authorization, Cystic Fibrosis, Vertex

Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.

Key Points: 
  • Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.
  • In these studies, the triple combination regimen was generally well tolerated.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Aradigm Announces Withdrawal of European Marketing Authorization Application (MAA) for Linhaliq

Retrieved on: 
Wednesday, October 30, 2019
Biotechnology, Pharmaceutical, Health, Bacteria, RTT, Gram-negative bacteria, Pseudomonadales, Medical specialties, Antibiotic-resistant bacteria, Pseudomonas aeruginosa, Cystic fibrosis, Bronchiectasis, Pseudomonas, Aradigm, Non-Cystic Fibrosis Bronchiectasis

Aradigm Corporation (OTCQB: ARDMQ) (Aradigm or the Company) today announced that following a recent Oral Explanation, it has withdrawn its Marketing Authorization Application (MAA) for Linhaliq as a treatment for non-cystic fibrosis bronchiectasis (NCFBE) patients with chronic lung infections with Pseudomonas aeruginosa (P. aeruginosa).

Key Points: 
  • Aradigm Corporation (OTCQB: ARDMQ) (Aradigm or the Company) today announced that following a recent Oral Explanation, it has withdrawn its Marketing Authorization Application (MAA) for Linhaliq as a treatment for non-cystic fibrosis bronchiectasis (NCFBE) patients with chronic lung infections with Pseudomonas aeruginosa (P. aeruginosa).
  • Information regarding Aradigms bankruptcy case and Aradigms Monthly Operating Reports may be obtained from the Bankruptcy Court.
  • Aradigm remains confident in the efficacy, safety and quality of Apulmiq (US) / Linhaliq (EMA).
  • Aradigm and the Aradigm Logo are registered trademarks of Aradigm Corporation.

Arrevus Secures Support from the North Carolina Biotechnology Center to Advance ARV-1801 for the Treatment of Pulmonary Exacerbations in Cystic Fibrosis Patients and Appoints Robert J. Schotzinger, M.D., Ph.D. to the Board of Directors

Retrieved on: 
Wednesday, October 30, 2019
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Other Health, RTT, Medical specialties, Organ systems, Channelopathies, Cystic fibrosis, Respiratory therapy, Breakthrough therapy, Idiopathic pulmonary fibrosis, Pulmonary fibrosis

The loan is intended to support the development of the companys Phase 3 drug candidate ARV-1801 for the treatment of pulmonary exacerbations in patients with cystic fibrosis.

Key Points: 
  • The loan is intended to support the development of the companys Phase 3 drug candidate ARV-1801 for the treatment of pulmonary exacerbations in patients with cystic fibrosis.
  • Pulmonary exacerbations are episodes of acute worsening of pulmonary status that occur frequently in patients with cystic fibrosis and result in a sequential and permanent decline in lung function.
  • Arrevus is excited to expand the available treatment options for patients with cystic fibrosis who so commonly have to contend with pulmonary exacerbations.
  • Arrevus also announced today the appointment of Robert J. Schotzinger, M.D., Ph.D. to the Arrevus Board of Directors.

Cystic Fibrosis Foundation Launches $500 Million Path to a Cure

Retrieved on: 
Wednesday, October 30, 2019
Research, Genetics, Clinical trials, Philanthropy, Other Health, Biotechnology, Pharmaceutical, Health, Foundation, Science, Medical specialties, Cystic Fibrosis Foundation, Venture philanthropy, Social economy, Mixed economies, Cystic fibrosis, Vertex Pharmaceuticals, Cystic Fibrosis Canada

Today the Cystic Fibrosis Foundation unveiled its Path to a Cure, an ambitious research agenda to deliver treatments for the underlying cause of the disease and a cure for every person with cystic fibrosis (CF).

Key Points: 
  • Today the Cystic Fibrosis Foundation unveiled its Path to a Cure, an ambitious research agenda to deliver treatments for the underlying cause of the disease and a cure for every person with cystic fibrosis (CF).
  • View the full release here: https://www.businesswire.com/news/home/20191030005196/en/
    The CF Foundation has a long track record of success in facilitating the development of new cystic fibrosis therapies.
  • The Path builds on the Foundations highly successful venture philanthropy strategy to stimulate industry investment in CF.
  • To learn more about research being funded under the Path to a Cure commitment and to follow progress on the multiple research efforts underway, please visit cff.org/PathtoaCure .

Petitions Committee welcomes deal on cystic fibrosis drugs

Retrieved on: 
Saturday, October 26, 2019
Cystic fibrosis, Drugs, Petitions Committee, Breakthrough therapy, Vertex Pharmaceuticals, Life sciences, Lumacaftor/ivacaftor, Health sciences, European Parliament Committee on Petitions, Paul Scully, Petition, Online petitions

Members of the Petitions Committee have welcomed todays announcement of a deal between the NHS and Vertex Pharmaceuticals to enable life-extending drugs for cystic fibrosis suffers to be available on the NHS.

Key Points: 
  • Members of the Petitions Committee have welcomed todays announcement of a deal between the NHS and Vertex Pharmaceuticals to enable life-extending drugs for cystic fibrosis suffers to be available on the NHS.
  • The announcement of a deal for access to Orkambi, Symkevi and Kalydec follows two petitions, which together secured more than 225,000 signatures and led to the Petitions Committee scheduling two popular debates on the issue.
  • The two petitions debated were:
    This petition gathered 118,034 signatures, and was debated on 19 March 2018
    Helen Jones MP, Chair of the Petitions Committee said:
    Todays deal is a welcome breakthrough for everyone who has tirelessly campaigned for this drug to be available to improve the lives of people with Cystic Fibrosis.
  • Paul Scully MP, a member of the Petitions Committee, said:
    This announcement is brilliant news for those living with Cystic Fibrosis.

Petitions Committee welcomes deal on cystic fibrosis drugs

Retrieved on: 
Saturday, October 26, 2019
Cystic fibrosis, Drugs, Petitions Committee, Breakthrough therapy, Vertex Pharmaceuticals, Life sciences, Lumacaftor/ivacaftor, Health sciences, European Parliament Committee on Petitions, Paul Scully, Petition, Online petitions

Members of the Petitions Committee have welcomed todays announcement of a deal between the NHS and Vertex Pharmaceuticals to enable life-extending drugs for cystic fibrosis suffers to be available on the NHS.

Key Points: 
  • Members of the Petitions Committee have welcomed todays announcement of a deal between the NHS and Vertex Pharmaceuticals to enable life-extending drugs for cystic fibrosis suffers to be available on the NHS.
  • The announcement of a deal for access to Orkambi, Symkevi and Kalydec follows two petitions, which together secured more than 225,000 signatures and led to the Petitions Committee scheduling two popular debates on the issue.
  • The two petitions debated were:
    This petition gathered 118,034 signatures, and was debated on 19 March 2018
    Helen Jones MP, Chair of the Petitions Committee said:
    Todays deal is a welcome breakthrough for everyone who has tirelessly campaigned for this drug to be available to improve the lives of people with Cystic Fibrosis.
  • Paul Scully MP, a member of the Petitions Committee, said:
    This announcement is brilliant news for those living with Cystic Fibrosis.

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.