The Anti-Infective Drugs Advisory Committee to the US Food and Drug Administration voted today in favor of the use of TIP by cystic fibrosis patients.

Researchers at the University of Iowa Carver College of Medicine have discovered a genetic process that can restore function to a defective protein, the most common cause of cystic fibrosis.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today final data from a Phase 2 study of VX-809 and KALYDECO™ (ivacaftor) that showed statistically significant improvements in lung function among adults with cystic fibrosis (CF) who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del.

PTC Therapeutics today announced the results from a Phase 3 study of ataluren that demonstrated positive trends in lung function and the rate of pulmonary exacerbations.

Gilead Sciences Inc. says it has made progress resolving a shortage of its inhaled antibiotic Cayston® that began earlier this year.

Inhaling concentrated saline mist does not reduce how often infants and young children with CF need antibiotics for respiratory symptoms, according to findings from a recent clinical trial.

Digestive Care, Inc. has received U.S. Food and Drug Administration approval of its new drug application for PERTZYE™, developed for the treatment of exocrine pancreatic insufficiency due to CF or other conditions.

Vertex Pharmaceuticals Inc. today announced promising interim results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ and VX-809, a CF drug in development.

PMD Healthcare is encouraging the cystic fibrosis community to enter a sweepstakes contest for a chance to win a personal spirometer.

The FDA has approved Ultresa, a new pancreatic enzyme product used to help aid food digestion in people with cystic fibrosis.

Gilead Sciences says it is working to address "significant and unanticipated challenges manufacturing enough Cayston to meet demand in the United States."

PMD Healthcare Inc. has launched the first and only personal spirometer that enables patients with lung diseases to easily and accurately monitor their lung function any time and anywhere.

Vertex Pharmaceuticals Inc. announced on January 31 that the FDA has approved KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis.

Vertex Pharmaceuticals is planning additional studies of KALYDECO™ in children with CF and in people with CF who have certain mutationsnot previously evaluated. Pending feedback from regulatory agencies, it plans to begin three studies in mid-2012.

According to the CF Foundation, an expanded access program for Kalydeco™ (VX-770) is now available at participating clinical sites for people with the G551D mutation who have highly limited lung function and may benefit from treatment.

Adding inhaled dry powder mannitol to standard CF therapy produced sustained improvement in lung function, according to a new study by Pharmaxis Ltd.

The FDA has granted a request by Vertex Pharmaceuticals for priority review of KALYDECO™, the first potential therapy to target the underlying cause of cystic fibrosis.

In addition to Type 1 and Type 2 diabetes, there is another variant of the disease: cystic fibrosis-related diabetes. It differs in some important ways from Type 1 and Type 2 diabetes and it requires different treatment methods.

The New England Journal of Medicine features a study this week about a Phase 3 clinical trial of Kalydeco™ (VX-770), a potential CF therapy undergoing review for approval by the U.S. Food and Drug Administration.

Vertex Pharmaceuticals today submitted a new drug application to the FDA for KALYDECO™ (VX-770). If approved, the drug will be the first treatment to target the underlying cause of cystic fibrosis.

Insmed Inc.today announced that it has been notified by the FDA that it is continuing the clinical hold previously placed on Insmed's phase 3 clinical trials for ARIKACE® in CF patients with Pseudomonas lung infections.

Biotech company Pulmatrix has decided to focus its efforts on PUR118, the company's lead clinical drug candidate for cystic fibrosis and chronic obstructive pulmonary disease.

The FDA has placed a clinical hold on phase 3 clinical trials for ARIKACE® in cystic fibrosis patients with Pseudomonas lung infections.

Vertex Pharmaceuticals Inc. said it is on track to submit in October a new drug application for VX-770, a promising CF therapy.

Aptalis Pharma announced that the FDA has approved a new strength of ZENPEP® (pancrelipase) Delayed-Release Capsules for exocrine pancreatic insufficiency.

The FDA has approved an infant-specific dose of Abbott's CREON® (pancrelipase) Delayed-Release Capsules to treat exocrine pancreatic insufficiency due to cystic fibrosis.

Vertex Pharmaceuticals announced a plan to provide VX-770, a cystic fibrosis medicine in development, to people with the G551D mutation who are in critical medical need and may benefit from treatment prior to potential approval of the drug from the U.S. Food and Drug Administration.

Vertex Pharmaceuticals reported positive final results of a Phase 3 clinical trial of VX-770, a CF medicine in development that targets the underlying cause of the disease.

Vertex Pharmaceuticals announced promising results from a Phase 2 study evaluating combinations of VX-770 and VX-809, potential medicines designed to treat the defective protein that causes cystic fibrosis.

Aradigm Corp. has received orphan drug designation from the FDA for ciprofloxacin for inhalation for the management of bronchiectasis.

A growing shortage of medications for a host of illnesses — including cystic fibrosis — has hospitals scrambling for substitutes to avoid patient harm, and sometimes even delaying treatment.

As part of its CF Awareness Month focus on exercise, the Boomer Esiason Foundation has released Episode 17 in the Cystic Fibrosis Wind Sprint series: Using the PowerLung.

WBUR's "Common Health" takes a closer look at the Vertex drug VX-770 in its recent special report, "New Cystic Fibrosis Drug Brings Gift of a Future."

The FDA has rejected Eli Lilly and Co.'s EPI drug candidate liprotamase and has ordered the company to conduct additional clinical trials on the potential therapy.

The CF Foundation is expandin its collaboration with Vertex Pharmaceuticals for the discovery and development of additional drugs aimed at treating the underlying cause of cystic fibrosis.

Read the complete Vertex Pharmaceuticals press release on its recent findings in the Phase 3 clinical trial of VX-770.

Vertex Pharmaceuticals reports positive results from Phase 3 clinical trial of VX-770 for children age 6 to 11.

Vertex Pharmaceuticals Inc. announced positive results from the Phase 3 STRIVE study of VX-770, a potential therapy that targets the defective protein that causes CF.

Axcan President and CEO Frank Verwiel explains how Axcan's merger with Eurand will affect customers.

An FDA advisory committee voted to recommend non-approval of liprotamase, currently under FDA review for the treatment of exocrine pancreatic insufficiency.

Mpex Pharmaceuticals, Inc., today announced it has initiated its Phase 3 clinical trial program with Aeroquin? (MP-376) for the treatment of pulmonary infections in patients with cystic fibrosis.

Inspire Pharmaceuticals, Inc. today announced disappointing results from its second Phase 3 clinical trial, TIGER-2, with denufosol tetrasodium for the treatment of cystic fibrosis.

PTC Therapeutics, Inc. announced today that it has completed enrollment of a Phase 3 clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF).

Inspire Pharmaceuticals, Inc. announced today that the results from its first Phase 3 clinical trial with denufosol tetrasodium for cystic fibrosis, TIGER-1, have been published in the American Journal of Respiratory and Critical Care Medicine.

In a study published in this week's New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase 2 clinical trial of 39 people with cystic fibrosis.

PTC Therapeutics, Inc. today announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine. The published data show that treatment with ataluren, an investigational new drug, resulted in statistically significant improvements in the production and function of cystic fibrosis transmembrane conductance regulator (CFTR), a critical protein lacking in CF patients.

Discovery Laboratories, Inc. today announced that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to Discovery's KL4 surfactant for the treatment of cystic fibrosis (CF). Orphan designation provides for up to seven years of U.S. market drug product exclusivity for the designated indication following marketing authorization.

Pharmaceutical company Pharmaxis today announced significant results of pooled data from its two large scale six month Phase III trials of Bronchitol (inhaled mannitol) in people with cystic fibrosis.

Gilead Sciences, Inc. announced that its head-to-head Phase III clinical trial of Cayston® (aztreonam for inhalation solution) versus tobramycin inhalation solution in cystic fibrosis patients with Pseudomonas aeruginosa achieved its co-primary endpoint of superiority of Cayston for mean actual change in forced expiratory volume in one second (FEV1, a measure of lung function) percent predicted across three treatment cycles (six months).

A new analysis of data from more than 12,000 people with cystic fibrosis (CF) and Pseudomonas aeruginosa (Pa) lung infection in the Cystic Fibrosis Foundation's Patient Registry shows that use of TOBI(R) (Tobramycin Inhalation Solution, USP) was associated with a 21% reduction in mortality in the following year. The results were presented today at the 24th Annual North American Cystic Fibrosis Conference (NACFC) in Baltimore, Maryland, sponsored by the Cystic Fibrosis Foundation.

Vertex Pharmaceuticals Inc. today announced the initiation of a Phase 2a clinical trial that will evaluate multiple combinations of VX-770 and VX-809, investigational oral cystic fibrosis transmembrane conductance regulator (CFTR) modulators for the treatment of cystic fibrosis. The trial is designed to evaluate the safety and tolerability of VX-809 dosed alone for 14 days followed by dosing of VX-809 in combination with VX-770 for 7 days. The trial will also assess the effect of VX-809 alone and in combination with VX-770 on CFTR function, as measured by sweat chloride. The three-part trial is designed to enroll up to a total of 160 people with two copies of the F508del CFTR mutation, the most common mutation in the CFTR gene.

PTC Therapeutics, Inc. announced a grant award of approximately $1.6 million from the U.S. Food and Drug Administration Office of Orphan Products Development to support an ongoing Phase 3 study of ataluren in patients with nonsense mutation cystic fibrosis.

Emily Schaller likes to travel as part of the cystic-fibrosis advocacy she does, but the daily regimen she uses to manage her disease weighs her down. "You have to bring three extra bags with meds and the vest," she said.

Eurand Pharmaceuticals, a subsidiary of specialty pharmaceutical company Eurand, has announced additional data from a post-hoc analysis of a Phase III clinical trial with Zenpep delayed-release capsules, an FDA-approved pancreatic enzyme product, or PEP, for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis or other conditions.

A 'best' hospital for CF kids: unhappy with its so-so record, Cincinnati Children's used tools of health reform to turn itself around.

A retrospective analysis of more than 1,500 patients suggests nothing is gained by extending intravenous (IV) antibiotic treatment beyond 8 to 10 days, or by giving antibiotics in the hospital.

Eli Lilly and Company and Alnara Pharmaceuticals, Inc. today announced they have signed a definitive merger agreement whereby Lilly will acquire Alnara, a privately held biotechnology company developing protein therapeutics for the treatment of metabolic diseases.

The CF Foundation has learned that the U.S. Food and Drug Administration will allow wholesalers to distribute the pancreatic enzyme product Pancrease® MT to pharmacies until July 12, 2010. At that time, an updated formula called PancreazeTM will become available and will replace Pancrease.

The Cystic Fibrosis Foundation has learned that the pancreatic enzyme product Pancrease will be available in limited supply at pharmacies around the country until an updated formula called Pancreaze is made available this summer.

Inspire Pharmaceuticals Inc. announced today that Inspire scientists will present data suggesting that denufosol, an inhaled ion channel regulator, has properties that allow it to reach and improve lung function in the small airways, which may support its potential as an early intervention therapy.

Mpex Pharmaceuticals, Inc. today announced the presentation of data from its Phase 2b clinical trial with Aeroquin that demonstrated statistically significant improvements in bacterial load, respiratory function and time to need for anti-pseudomonal antibiotics versus placebo in a heavily treated patient population.

Children and adolescents with cystic fibrosis who received the antibiotic azithromycin did not experience improved lung function, compared with patients who received placebo, according to a study published in the May 5 issue of the Journal of the American Medical Association.