Cystic Fibrosis: Update Bulletin #3

  • ID: 4521375
  • Newsletter
  • FirstWord Publishing
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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:
  • How do KOLs perceive Boehringer Ingelheim’s adalimumab biosimilar, Cyltezo?
  • With three adalimumab biosimilars now approved in Europe, what concerns do prescribers have with regard to multiple biosimilar versions becoming available?
  • 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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