While Orphazyme's attempts to get approval for arimoclomol failed miserably, new owner KemPharm is confident it can get the oral heat shock protein amplifier across the regulatory finishing line for Niemann-Pick disease type C.

Public Company Edition: Applied Molecular Transport, Scholar Rock, Agios and others join many of their peers in revising their strategies and downsizing their teams. Also, Zentalis grossed $200m from a follow-on offering and OKYO Pharma went public in the year’s smallest biopharma IPO in the US. 

Saniona and Bone Therapeutics are the latest companies on the continent to be forced into drastic restructuring, shedding jobs and research projects to preserve rapidly dwindling cash piles.

The European Medicines Agency’s human medicines committee has recommended five new drugs for use in the EU at its latest monthly meeting.