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Cystic Fibrosis: Update Bulletin [December 2017]

Product Code:
Publication Date:
December 2017

This edition presents world leading key opinion leader (KOL) views on recent developments in the cystic fibrosis (CF) space. Topics covered include; expert opinions on Vertex Pharmaceuticals’ regulatory submission to the FDA and EMAfor tezacaftor/ivacaftor, a second-in-class cystic fibrosis transmembrane conductance regulator (CFTR), which has been granted Priority Review status in the US;  ProQR Therapeutics’ publication of positive preliminary results from a Phase 1b study assessing QR-010,a novel investigational RNA therapeutic with Orphan Drug status in the EU and US and Fast Track review status granted by the FDA; and Savara’s initiation of a Phase III study with AeroVanc (inhaled vancomycin powder) for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA), which is an increasing problem amongst CF patients.

Business Questions:

• In August 2017, Vertex Pharmaceuticals reported that the FDA and the EMA had accepted the regulatory submission for tezacaftor/ivacaftor, but how do KOLs perceive the company’s next generation cystic fibrosis transmembrane conductance regulator (CFTR)?
• How does tezacaftor/ivacaftor compare with its earlier launched competitor Orkambi (lumacaftor/ivacaftor)?
• What impact will tezacaftor/ivacaftor have on the current treatment paradigm? Which subgroups of CF patients will be considered eligible candidates for this premium priced targeted therapy?
• What barriers to market entry exist for Vertex’s tezacaftor/ivacaftor?
• How promising is ProQR’s first-in-class investigational RNA therapeutic QR-010?
• What is the potential patient population for this highly targeted therapy? How will it compete with the CFTR therapies?
• Do KOL’s consider QR-010’s inhaled route of administration to be a potential issue in the treatment of CF?
• Savara announced progression of its inhaled vancomycin powder, AeroVanc, to Phase III development for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA), but how positive are KOLs with regard to this product given that it’s available generically in other formulations?
• How significant is the issue of MRSA amongst CF patients? How will AeroVanc sit within the current treatment paradigm and what products, if any, will it displace?
• What factors, if any, will impact AeroVanc’s uptake for the intended indication?


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