ORKAMBI (lumacaftor / ivacaftor), CFTR gene corrector and potentiator

• ORKAMBI has received a Marketing Authorisation for the treatment of cystic fibrosis in children aged 12 years and older, homozygous for the F508del mutation of the CFTR gene.
• The efficacy of ivacaftor, in combination with lumacaftor, was demonstrated short-term (24 weeks) despite an open-label follow-up until 48 weeks, on an interim endpoint (absolute change in FEV1 value). The improvement observed compared to placebo is modest (3 to 4% depending on the studies).
• The efficacy of this combination on long-term disease progression is unknown. The lack of long-term morbidity, mortality and safety data do not allow the benefit of the medicinal product to be assessed in the overall management and progression of the disease, in particular on the progression of lung bacterial colonization and its resistance to antibiotics.
• The main adverse effects observed were dyspnoea, diarrhoea and nausea.

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