The U.S. Food and Drug Administration (FDA) has granted the designation of orphan drug to KB407, Krystal Biotech’s potential inhaled gene therapy for the treatment of cystic fibrosis (CF). “We are pleased to receive Orphan Drug Designation for KB407 to treat cystic fibrosis as…
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The nonprofit Claire’s Place Foundation has launched a pilot program to help 15 adults with cystic fibrosis (CF) or caregivers of patients acquire flexible employment, enabling them to continue working while dealing with the disease’s daily needs. The Work Proudly Program aids people in choosing careers and gaining the…
AzurRx BioPharma has enrolled the first three patients in its Phase 2b trial investigating MS1819 as a treatment for exocrine pancreatic insufficiency (EPI) in cystic fibrosis (CF) patients. The trial, dubbed OPTION 2 (NCT04375878), aims to enroll 30 CF patients ages 18 and older. Top-line…
MS1819, AzurRx BioPharma’s investigational treatment for exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF), safely increased fat absorption in the first five patients receiving the therapy alongside pancreatic enzyme replacement therapy (PERT) in a Phase 2 clinical trial. The interim analysis also showed the…
A Phase 1/2 clinical trial assessing Arrowhead Pharmaceuticals‘ investigational inhaled therapy, ARO-ENaC, for the treatment of cystic fibrosis (CF), has dosed its first participants, the company announced. The AROENaC1001 trial (NCT04375514) is designed to investigate ARO-ENaC in 24 healthy volunteers and 30 CF patients, and is…
Cystic Fibrosis Ireland has set aside up to €120,000 ($141,150) to provide personal protective equipment (PPE) to people with cystic fibrosis (CF) in the Republic of Ireland. Those interested may apply for individual grants of €100 per applicant through Aug. 22. Applications to the PPE grant should be…
New research unveils why the bacteria Pseudomonas aeruginosa cause sustained infections in younger cystic fibrosis (CF) patients, while the bacteria Burkholderia cepacia complex infects patients as teenagers and adults. The findings show that P. aeruginosa loses the ability to use its toxic weaponry after it adapts to the lungs, allowing Burkholderia cepacia complex to…
The Cystic Fibrosis Foundation has committed $20 million to support the development of innovative therapeutics and early-stage companies focusing on cystic fibrosis (CF). In collaboration with venture capital firm Longwood Fund, the funding is part of the foundation’s Path to a Cure, a $500 million initiative…
A national newborn screening (NBS) program in the Republic of Ireland suggests that the incidence of cystic fibrosis and CF carriers in the country is lower than previously estimated, a study found. It also shows that the Gly551Asp mutation is almost three times more common among newborns in Ireland…
The Scottish Government and Vertex Pharmaceuticals have reached a deal to make Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for people with cystic fibrosis (CF) once the medication receives its European license. This follows similar agreements in the three other U.K. countries, England, Wales, and Northern…
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