Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
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Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
Dr. Frank Accurso, director of cystic fibrosis clinical research at Children's Hospital Colorado and University of Colorado Denver, participated in a congressional roundtable discussion at National Jewish Health in Denver, Colorado.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
The Cystic Fibrosis Foundation invited more than 100 scientists to its research conference in Savannah, Ga., last month to discuss recent advancements in gene therapy, gene editing and stem-cell biology and how these new technologies could be applied to finding a one-time cure for cystic fibrosis.
The Associated Press published a story this week highlighting the Cystic Fibrosis Foundation's "groundbreaking" drug development model and its role in developing CF treatments that target the root cause of the disease.
The Cystic Fibrosis Foundation has entered into an agreement with Synspira Therapeutics Inc. to develop a non-porcine enzyme replacement therapy to offer an alternative to people with cystic fibrosis who cannot digest food properly.
At the 2016 North American Cystic Fibrosis Conference (NACFC) held on Oct. 27-29 in Orlando, Fla., the Cystic Fibrosis Foundation announced the recipients of seven awards given to members of the research and medical community whose achievements have helped advance cystic fibrosis research and care.
The Cystic Fibrosis Foundation recently hosted the CFF Research Conference: Pushing the Frontiers, bringing together nearly 150 basic scientists, clinical researchers, graduate students and pharmaceutical company representatives to share information on recent advances and critical challenges in CF drug discovery and development.