The approval was based on data from a phase 3 study that evaluated Trikafta in 66 children 6 to 11 years of age with CF who were either homozygous for the F508del mutation or heterozygous for the F508del mutation and 1 minimal function mutation.
The Pulmonary Clinical Practice Guidelines Committee of the Cystic Fibrosis (CF) Foundation provided updated recommendations on the use of available chronic treatments for Cystic Fibrosis based on current evidence.
Bronchitol should only be used by adults who have passed the Bronchitol Tolerance Test.
The full list of CFTR gene mutations for Trikafta, Symdeko and Kalydeco can be found in the updated prescribing information for each respective product.
Bronchitol is only indicated for use in adults who have passed the Bronchitol Tolerance Test.
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.
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 inhibited the therapeutic benefit of the cystic fibrosis transmembrane conductance regulator modulators in pediatric patients.
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.
The FDA has granted Orphan Drug designation to ARV-1801 (sodium fusidate; Arrevus) for the treatment of pulmonary exacerbations in patients with cystic fibrosis.
