October Vertex Update


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.

Kalydeco Availability
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)

2. R117H

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ProQR Receives Orphan Drug Status for F508del Therapy

More hope on the horizon for those with cystic fibrosis. New drug in development.


ProQR Therapeutics recently received EMA and FDA orphan drug designation for their therapy targeting Cystic Fibrosis. ProQR Therapeutics are developing an antisense oligonucleotide that targets the F508del Cystic Fibrosis mutation. This is currently in the pre-clinical stage.

This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein.CF gene mutations are present in the DNA, this defect is copied into the RNA, which is copied into the protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level. This means the normal CFTR protein is made from the RNA, restoring chloride transport.

Sources:EMA COMP September 2013 Report andFDA Orphan Drug Designation

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