Kalydeco: New data with G551D
Data from the PERSIST study showed that after 144 weeks of Kalydeco there was a durable treatment effect in FEV1, weight and other measures. Side-effects were consistent with previous studies. Data from the GOAL study showed a lower rate of hospitalisations, improved intestinal function and decreased bacterial colonisation.
Currently available in England, Scotland, Northern Ireland, Wales, the Republic of Ireland, France, Germany, the Netherlands, Austria, Denmark, Sweden, Norway, Greece and the US.
‘Vertex is in active discussions with relevant agencies in Australia and Canada regarding public reimbursement of KALYDECO in these countries.’
Kalydeco Label Expansion
1. Gating Mutations
– Application submitted to FDA (US) and EMA (Europe) for people with CF aged 6 and older with a non G551D gating mutation
– Approx 400 people have a non G551D gating mutation in North America, Europe and Australia (aged 6 and older)
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