The sponsors of four drugs were due or possibly due to appear before the European Medicines Agency to explain why their respective products merit EU-wide approval.  

AstraZeneca is preparing a US filing for hereditary transthyretin-mediated amyloid polyneuropathy for eplontersen, which it hopes will compete with Pfizer and Alnylam's therapies in that rare disease space.

The market for new company creation in the biopharma sector remains healthy and poised to continue fostering the establishment of start-ups, based on a new In Vivo study of the strong initial investments that pharma and biotech companies receive.

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.