News

The U.S. approval of CFTR modulators for cystic fibrosis (CF) may have reduced or delayed the need for liver transplants among people in the country with CF-related liver disease, or CFrLD, a new study suggests. Since the U.S. Food and Drug Administration (FDA) approved Trikafta (elexacaftor/tezacaftor/ivacaftor) — the first…

Researchers in Italy have developed a new experimental compound called 3b, which has shown early promise as a dual-action therapy for cystic fibrosis (CF). It addresses the dysfunction of the CFTR protein that causes the disease, as well as protects against viral threats that can worsen lung problems. In…

The U.S. Food and Drug Administration (FDA) has approved a new generic form of inhaled tobramycin, an antibiotic that’s used to treat bacterial lung infections in people with cystic fibrosis (CF). The newly approved therapy, tobramycin inhalation solution, will be sold by The Ritedose Corporation through its…

Vertex Pharmaceuticals presented new data highlighting additional clinical benefits and improved outcomes in people with cystic fibrosis (CF) treated with the triple combination Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) or other CFTR modulators. The data also suggest that Alyftrek may lead to fewer pulmonary exacerbations (flare-ups) and less antibiotic usage than…

Sionna Therapeutics has launched a clinical trial to test its experimental therapy SION-719 in combination with Trikafta (elexacaftor/tezacaftor/ivacaftor), which is currently the standard-of-care treatment for cystic fibrosis (CF). According to a company press release, the Phase 2a study (NCT07108153) will enroll adults with CF who…

ARCT-032, Arcturus Therapeutics’ experimental inhaled therapy for cystic fibrosis (CF), has been well tolerated so far in a clinical trial. Early imaging data also suggest it may help clear mucus from the lungs, according to interim results announced by the company. “These findings suggest that ARCT-032 may be…

Despite a recent hold by the U.S. Food and Drug Administration (FDA) on a BX004 clinical trial in the U.S., the experimental cystic fibrosis (CF) lung infection treatment is still advancing in Europe. According to BX004’s developer Biomx, top-line results are expected in early 2026. “Patient enrollment and…

Enrollment has finished in a clinical trial testing the experimental therapy CMTX-101, which is designed to help clear out bacterial lung infections in people with cystic fibrosis (CF) and other diseases. Top-line results from the Phase 1b/2a study (NCT06159725) are expected to be announced in the first quarter…

The Cystic Fibrosis Foundation is making a new investment of up to $11 million in 4D Molecular Therapeutics (4DMT) to support the development of 4D-710, the company’s inhaled gene therapy for cystic fibrosis (CF) that is currently in clinical development. The funding, which builds on earlier support,…

While treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) helps people with cystic fibrosis (CF) gain weight, a tendency toward unhealthy diets may pose new risks for patients — warranting “close monitoring and interventions to prevent excessive weight gain” — a study from Norway reports. People with CF in the European nation who…