Interview with Mark Rothera, PTC Therapeutics

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Freya Smale

An Interview with Mark Rothera, Chief Commercial Officer, PTC Therapeutics

We are thrilled that Mark is once again joining us at this year’s World Orphan Drug Congress USA.  We interviewed Mark Rothera, Chief Commercial Officer of PTC Therapeutics to share with us insights on their journey. We hope this helps and encourages all the other pharma and biotech companies developing orphan drugs for patients with significant unmet needs to continue on with determination.

Game-changer Translarna for over 2,000 rare diseases with a nonsense mutation

Translarna is an orphan drug for nonsense mutation Duchenne Muscular Dystrophy which is now approved in Europe, Israel and Korea. “It was an 18-year journey to bring the first treatment to patients with DMD due to a nonsense mutation, with many bumps in the road; but it was all worth it”.

Download this eBook now to find out more about:

  • Game-changer Translarna for over 2,000 rare diseases with a nonsense mutation. What makes Translarna unique?
  • What’s next for Translarna after the latest Phase 3 clinical trials
  • Reactions to Biomarin’s previously GSK/Prosensa’s orphan drug disappointing Ph 3 study results and the pending Sarepta results
  • How to choose which to prioritize from among the 2,000 rare diseases with a nonsense mutation
  •  The most challenging part in getting approval in Europe
  • How early collaboration with regulators should be
  • What processes or steps helped along this regulatory collaboration and approval
  • Market access
  • At what point would PTC sell

And of course a sneak preview of Mark’s keynote session at the World Orphan Drug Congress USA next April.

Get your copy here >

Mark is speaking at this year’s World Orphan Drug Congress USA on “Targeting ‘bigger’ targets: increase nonsense mutation read through applicable to other rare diseases.” 

Join him at the event by registering today!

World Orphan Drug Congress USA 2016