News

Alyftrek, triple-combination CF treatment, approved in UK

The U.K. Medicines and Healthcare Products Regulatory Agency approved Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) as a cystic fibrosis (CF) treatment for patients ages 6 and older. The triple-combination CFTR modulator, developed by Vertex Pharmaceuticals, is indicated for patients who have at least one copy of a responsive…

Kaftrio eases some CF symptoms after lung transplant: Study

Kaftrio, a combination of elexacaftor, tezacaftor, and ivacaftor that’s marketed as Trikafta in the U.S., may ease breathing and digestive symptoms in adults with cystic fibrosis (CF) who’ve had a lung transplant, a study from the Netherlands suggests. Researchers did observe a slight, but significant, increase in creatinine,…

Biomx raises $12M to advance phage therapy for CF infections

Biomx has raised $12 million in financing to advance BX004, a phage therapy being developed to treat bacterial infections — specifically, ones caused by Pseudomonas aeruginosa, known as P. aeruginosa for short — in people with cystic fibrosis (CF). The funding comes from investors purchasing the company’s stock,…

EMA panel recommends Kaftrio for range of CF-causing mutations

A European Medicines Agency (EMA) committee recommended that the label for Kaftrio be expanded to cover treatment for people with cystic fibrosis (CF) caused by a wider array of mutations, Kaftrio’s developer Vertex Pharmaceuticals said. The Committee for Medicinal Products for Human Use (CHMP) opinion will be reviewed…

CF treatment Orkambi shows 2-year benefits in real-world study

Starting treatment with the CFTR modulator Orkambi (lumacaftor/ivacaftor) early in life may provide long-term benefits for children with cystic fibrosis (CF), yet lung function improvements may be temporary, according to a real-world study from Hungary. The study, “Body composition changes and clinical outcomes in pediatric cystic fibrosis…

Baby gut bacteria fails to mature normally with CF, study finds

Young children with cystic fibrosis (CF) have less mature gut bacterial growth over time than do their typically developing peers, according to a new U.S. study. “Our findings demonstrate that the gut microbiota of infants with CF fails to undergo typical developmental maturation, instead remaining entrenched in a transitional-like…