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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.
Eloxx Pharmaceuticals Provides Enrollment Update for Ongoing Phase 2 Clinical Studies for Cystic Fibrosis firstwordpharma
July 07, 2021
Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today provided an update on enrollment for 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.
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.
Vertex Announces U.S. FDA Approval for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations drugs
June 10, 2021
Vertex Pharmaceuticals Incorporated today announced the FDA approved expanded use of Trikafta to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance
First-of-Its-Kind Study Uses Smartphone to Track Cystic Fibrosis in Real Life firstwordpharma
June 03, 2021
Today, the Cystic Fibrosis Foundation announced the initiation of the HERO-2: Home Reported Outcomes Study, a first-of-its-kind, at-home observational study that aims to characterize the range of outcomes people experience while using Trikafta®
Australian TGA approves Vertex’s Trikafta for cystic fibrosis treatment pharmaceutical-technology
March 26, 2021
The Australian Therapeutic Goods Administration (TGA) has approved the use of Vertex Pharmaceutical’s Trikafta (elexacaftor, tezacaftor, ivacaftor and ivacaftor) for people with cystic fibrosis (CF) aged 12 years and above.
MHRA approves Polyphor’s phase 1 trial of novel inhaled antibiotic pharmatimes
December 23, 2020
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorised Swiss biotech Polyphor’s first-in-human phase I study of its novel inhaled antibiotic murepavadin.
Vertex’s Kalydeco scores another label extension in EU pharmatimes
November 09, 2020
Vertex has scored approval from the European Commission to extend the label for Kalydeco (ivacaftor) to include the treatment o infants with cystic fibrosis (CF) aged four months or older.

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