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Latest From Orphazyme A/S
Following Orphazyme’s disappointment in the US regarding its investigational drug for an ultrarare disease, the company has said it will need more time to address outstanding issues raised by EU reviewers about the same medicine.
When Breakthrough Falls Short: US FDA Complete Response Letters Rising Among BTD Applications For Approval
More than half of the complete response letters issued for applications holding breakthrough status have been issued since 2020, reflecting difficult – and often rancorous – efficacy debates.
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.
Following a week of "insane trading" which saw the Danish stock soar based on social media chatter, US regulators have surprised the firm and investors by issuing a complete response letter for arimoclomol for Niemann-Pick disease type C.
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