Maureen Newman,  —

Aptalis Pharma continued to evaluate its pancreatic enzyme products (PEPs) in patients with cystic fibrosis and exocrine pancreatic insufficiency in its Phase 4 clinical trial of Panzytrat® 25,000. Results show that patients taking Panzytrat had better control over their steatorrhea, or fecal fat content. The study was designed to…

After the Food and Drug Administration (FDA) issued rules requiring approval of pancreatic enzyme products (PEPs) in 2004, the cost and availability of the products added another hardship to the lives…

A number of factors influence the health-related quality of life of patients with cystic fibrosis. A new study in the Italian Journal of Pediatrics written by a collaboration of researchers…

Vertex Pharmaceuticals‘ two-part proof-of-concept study evaluating ivacaftor (under the name KALYDECO) in 24 cystic fibrosis patients shows data consistent with previous preclinical observations. An eight-week open-label period resulted in improved lung function in patients, initiating plans for a future Phase 3 trial. Each of the 24…

A group of thirteen scientists at the University of North Carolina School of Medicine may know why certain cystic fibrosis treatments improve lung function. Their answer: mucins, the proteins within mucus. “Our finding suggests that diluting the concentration of mucins in cystic fibrosis mucus is a key to better…

PTC Therapeutics, Inc. announced unexpected, yet encouraging results of their product ataluren. The biopharmaceutical company focuses on developing small-molecule drugs that target post-transcriptional control processes to alter protein production. Ataluren is PTC’s investigational new drug designed to restore protein function in cystic fibrosis patients with a nonsense mutation (nmCF)…

Lexington, Massachusetts-based Pulmatrix made an impact at the 2014 Respiratory Drug Delivery Conference being held in Fajardo, Puerto Rico. A poster from the biotechnology company presented data related to PUR0400 (fluoroquinilone Levofloxac), a preclinical drug candidate formulated using Pulmatrix’s proprietary iSPERSE™ dry powder technology. PUR0400 is a drug formulation that…

Adding to the excitement of Vertex Pharmaceuticals Inc.‘s new orphan drug status of VX-661 is the announcement of the results of Vertex’s Phase 2 clinical study of…

Vertex Pharmaceuticals continues to break new ground on developing treatments for the orphan disease cystic fibrosis. Most recently, Vertex received the FDA’s Orphan Drug Designation for its investigational drug VX-661. Orphan drug designation was established in January 1983 as a result of the Orphan Drug Act in order to encourage…

An antimicrobial peptide produced by human immune cells may actually promote bacterial survival in cystic fibrosis patients with a chronic lung infection of Pseudomonas aeruginosa and lead to respiratory failure. The discovery was made by Daniel Wozniak and colleagues from Ohio State University Wexner Medical Center, who published a…