Orphan Diseases Report

25 September 2018

This report includes news and forward-looking analysis on 13 drugs in development across nine orphan indications.

A sampling of headlines:

• Alexion’s ravulizumab has potential to replace Soliris in aHUS, but biosimilar and other market competition looms, experts say
• Achillion’s Phase II C3 glomerulopathy trial of ACH4471 likely to be impacted by slow recruitment though experts optimistic about final results
• Ultragenyx faces dicey proposition for FDA filing sans Phase III LC-FAOD trial for triheptanoin but potential allowance based on orphan status, Phase II data, experts say