News

ELX-02 Plus Kalydeco Fails to Reach Trial’s Efficacy Goals

Treatment with ELX-02 in combination with Kalydeco (ivacaftor) was well-tolerated, but failed to meet efficacy goals in a clinical trial that tested the combo in people with cystic fibrosis (CF) caused by nonsense mutations. Eloxx Pharmaceuticals, the company developing ELX-02, says the lack of efficacy may…

Acne Seen as Adverse Effect of Trikafta Treatment in Case Study

Acne might be a side effect of Trikafta therapy in cystic fibrosis (CF) patients, according to a new case series in the U.S. Trikafta (elexacaftor, tezacaftor, ivacaftor) is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator that helps the defective CFTR protein function more effectively in CF…

Kaftrio, Trikafta in US, Safely Helps Woman Through Pregnancy

Use of Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold in the U.S. as Trikafta, helped a woman with cystic fibrosis (CF) more easily become pregnant and maintain her health throughout the pregnancy and child’s birth, according to a case report from France. The woman had struggled in a previous pregnancy, due to…

Supplemental Oxygen May Alter Lung Microbiome in CF Patients

Supplemental oxygen lessened the abundance of some microbial species in sputum samples from people with cystic fibrosis (CF) while sparing others, including Pseudomonas aeruginosa and Staphylococcus aureus, the main causes of bacterial lung infections in patients, a study reported. A need for caution exists when using supplemental oxygen, “historically ……

New Adrulipase Formulation Slated for Proof-of-concept Phase 2 Trial

First Wave BioPharma has developed a new optimized formulation of adrulipase, a yeast-derived enzyme designed to treat exocrine pancreatic insufficiency in people with cystic fibrosis (CF). The new formulation ensures that the enzyme is packed in acid-resistant micro-granules and protected from the acid environment of the stomach. The enzyme then reaches…

CF Foundation Joins CEO Action for Diversity and Inclusion

The Cystic Fibrosis Foundation (CFF) has joined the CEO Action for Diversity and Inclusion, adding its name to the more than 2,300 corporate leaders, representing 85 industries, who have pledged to bring greater inclusion and diversity into their workplaces. Organizations with diverse teams perform better, the CFF stated…