Orphazyme accelerates arimoclomol pre-launch activities and updates financial outlook for 2020

Orphazyme A/S
Company announcement                                                                                       
No. 67/2020                                                                                                          
Company Registration No. 32266355

Copenhagen, Denmark, October 23, 2020 – Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein (HSP) response for the treatment of neurodegenerative orphan diseases, today announces an update to its financial outlook for 2020. The update is primarily driven by an acceleration in commercial and other pre-launch activities during the fourth quarter of 2020 in preparation for potential approval of its investigational product candidate arimoclomol in Niemann-Pick disease Type C (NPC), which is currently under Priority Review by the U.S. Food and Drug Administration (FDA) with a target action date of March 17, 2021.

The increased expenditure is based on Orphazyme’s reinforced financial position following a successful global offering, as well as the FDA acceptance of its New Drug Application (NDA) for arimoclomol for NPC and granting of Priority Review. The Company also plans additional investments to further develop its pipeline.    

Kim Stratton, Chief Executive Officer of Orphazyme, said, “There is real momentum here at Orphazyme as we move closer to potential approval of arimoclomol in the U.S. in its first indication of NPC and accelerating our preparatory efforts now will help ensure a smooth launch. There are currently no approved products for NPC in the US and arimoclomol has the potential to make a significant difference to patients with this devastating disease, so our team is working expeditiously to ensure we are optimally positioned for a successful launch if approved.”