UBC Faculty Research and Publications

New drug for cystic fibrosis : Regulatory approval, clinical uncertainty? University of British Columbia. Therapeutics Initiative


Therapeutics Letter 116 examines a new drug therapy for cystic fibrosis (CF). Conclusions: Lumacaftor/ivacaftor (Orkambi) is a new combination drug for the treatment of CF. Health Canada granted regulatory approval for this drug combination based on two 24-week placebo-controlled studies showing a 3% improvement with the drug in a lung function test (ppFEV1). There is insufficient evidence at the present time that lumacaftor/ivacaftor improves quality of life, morbidity or mortality in patients with cystic fibrosis.

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