Kalydeco Approval Expanded to Include Younger Cystic Fibrosis Patients
The approval was based on data from a phase 3 open-label safety study that included a cohort of 6 children 4 months to <6 months of age.
Cystic Fibrosis
Pravibismane Gets Orphan Drug Status for Pulmonary Infections in Cystic Fibrosis
The FDA has granted Orphan Drug designation to pravibismane suspension for inhalation (BisEDT antimicrobial suspension; Microbion) for the management of pulmonary infections in patients with cystic fibrosis.
Tobacco Smoke Exposure Limits Benefits of CFTR Modulators in Cystic Fibrosis
Tobacco smoke exposure inhibited the therapeutic benefit of the cystic fibrosis transmembrane conductance regulator modulators in pediatric patients.
Inhaled Messenger RNA Treatment Fast-Tracked for Cystic Fibrosis
The FDA has granted Fast Track designation to MRT5005 (Translate Bio), an inhaled messenger RNA (mRNA) therapeutic being investigated for the treatment of cystic fibrosis.
Investigational Antibiotic Gets Orphan Drug Status for Pulmonary Exacerbations of Cystic Fibrosis
The FDA has granted Orphan Drug designation to ARV-1801 (sodium fusidate; Arrevus) for the treatment of pulmonary exacerbations in patients with cystic fibrosis.
New Drug Product: Trikafta
This slideshow shows drug information for Trikafta. Click here for the complete Trikafta monograph.
Controlling Antibiotic Adverse Drug Reactions in Cystic Fibrosis
The majority of patients with cystic fibrosis experience at least 1 antibiotic adverse drug reaction.
Elexacaftor-Tezacaftor-Ivacaftor Efficacious for Cystic Fibrosis
Percentage of FEV1 higher at four weeks and through 24 weeks with treatment versus placebo
MPR Weekly Dose Podcast: Episode 10
This week we talk about the resurrection of Biogen’s novel Alzheimer treatment; A “landmark” approval in cystic fibrosis; Expanded indication for Farxiga; The first topical minocycline formulation for acne vulgaris; and the Johnson & Johnson baby powder recall.
FDA Approves Triple Combination Therapy for Cystic Fibrosis
The Food and Drug Administration (FDA) has approved Trikafta (elexacaftor/tezacaftor/ivacaftor; Vertex) for the treatment of patients aged 12 years and older with cystic fibrosis (CF) who have ≥1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Trikafta is a fixed-dose combination containing elexacaftor 100mg, tezacaftor 50mg, and ivacaftor 75mg that is co-packaged…
