PTC Therapeutics Initiates Confirmatory Phase 3 Clinical Trial of Translarna™ (ataluren) in Patients with Nonsense Mutation Cystic Fibrosis

PTC Therapeutics, Inc. today announced the initiation of a global confirmatory Phase 3 clinical trial of Translarna™ (ataluren), an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations within cystic fibrosis are categorized as Class I mutations, … Continued

Positive results from Vertex F508del Phase 3 Study with Lumacaftor in Combination with Ivacaftor

Two 24-Week Phase 3 Studies of Lumacaftor in Combination with Ivacaftor Met Primary Endpoint with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation Combination of lumacaftor and ivacaftor … Continued

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels

BACKGROUND: The most common cystic fibrosis-associated mutation, the deletion of phenylalanine 508 (F508del), results in channels with poor membrane expression and impaired function. VX-770, a clinically approved drug for treatment of CF patients carrying the G551D mutation, and VX-809, a … Continued

CURx Pharmaceuticals Announces License Agreement with Gilead Sciences

CURx Pharmaceuticals today announced it has entered into a global license agreement with Gilead Sciences, Inc. for development of Fosfomycin:Tobramycin for Inhalation (FTI) to treat Pseudomonas aeruginosa lung infection in cystic fibrosis (CF) patients. FTI is ready for Phase III clinical trials, having successfully completed … Continued

Insmed Provides Interim Update from Two-Year, Open-Label Extension Study of ARIKACE

Insmed Incorporated, a biopharmaceutical company focused on developing an inhaled anti-infective to treat patients battling serious lung diseases in orphan indications that are often life-threatening, provides an interim update from the Company’s CLEAR-110 study, an ongoing, two-year, open-label extension study of once-daily ARIKACE®, … Continued

Arikace to Receive Orphan Medicinal Product Designation in the European Union to Treat Lung Infections Caused by Nontuberculous Mycobacteria

Insmed Incorporated, a biopharmaceutical company focused on developing an inhaled anti-infective to treat patients battling serious lung diseases in orphan indications that are often life-threatening, today announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued … Continued

Vertex Announces Recent Progress and Upcoming Milestones in Research and Development Programs for Cystic Fibrosis

Enrollment complete for TRAFFIC and TRANSPORT Phase 3 studies of lumacaftor (VX-809) in combination with ivacaftor for people with two copies of the F508del mutation (homozygous); data expected in mid-2014 12-week Phase 2 study of VX-661 in combination with ivacaftor … Continued