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A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations July 20, 2021
An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations.
Opportunities remain to address unmet needs in cystic fibrosis therapeutics market: GlobalData expresspharma
July 05, 2021
CFTR modulators are currently the only available treatments that target the underlying cause of CF. However, some patients with rare CFTR mutations are still ineligible to receive currently marketed therapies.
Unmet needs and opportunities in the cystic fibrosis market pharmaceutical-technology
July 05, 2021
The cystic fibrosis (CF) therapeutics market has a considerable number of unmet needs. GlobalData’s upcoming CF report indicates key unmet needs in this space including the lack of curative therapies, low patient adherence to treatment, limited choice ...
Scientists Demonstrate Promising New Approach for Treating Cystic Fibrosis firstwordpharma
June 17, 2021
UNC School of Medicine scientists led a collaboration of researchers to demonstrate a potentially powerful new strategy for treating cystic fibrosis (CF) and potentially a wide range of other diseases.
EC approves expanded indication for cystic fibrosis treatment europeanpharmaceuticalreview
December 12, 2019
The European Commission has approved the label extension for KALYDECO® (ivacaftor) to include the treatment of infants with cystic fibrosis between six and 12 months old.
FDA Approves Symdeko (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in the CFTR Gene drugs
June 24, 2019
FDA Approves Symdeko (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in the CFTR Gene.
Proteostasis Therapeutics Announces Global License Agreement with Genentech americanpharmaceuticalreview
December 18, 2018
Proteostasis Therapeutics announced a worldwide, exclusive license agreement with Genentech, a member of the Roche Group...
Vertex’s cystic fibrosis drugs become publicly available in Denmark pharmaceutical
November 19, 2018
Vertex Pharmaceuticals has signed an access agreement with Denmark’s pharmaceutical procurement agency Amgros for public hospital use of all current and future drugs for mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
AbbVie assumes full development and commercial control of CF programme pharmatimes
November 01, 2018
AbbVie has announced that it will assume full development and commercial responsibility for its collaboration with Galapagos to discover and develop new therapies to treat cystic fibrosis (CF).
Vertex announces reimbursement agreement in Australia for ORKAMBI biospectrumasia
September 29, 2018
Australians with CF join thousands of patients worldwide who are already receiving lumacaftor/ivacaftor