EMA's Advisory Committee Does Not Back Orphazyme's Arimoclomol Application

Orphazyme A/S ORPH has been notified by the Committee for Medicinal Products for Human Use (CHMP) of a negative trend vote on arimoclomol application following an Oral Explanation.

• The application covers arimoclomol for Niemann-Pick disease type C (NPC), a rare genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. 
• The Company said that it was encouraged by the ad-hoc expert group meeting's positive feedback on February 17, 2022.
• Related: Orphazyme Tightens FY21 Revenue Outlook.
• But the trend vote indicates that the CHMP's current orientation is to not approve arimoclomol when it convenes by the end of March 2022. 
• Orphazyme considers it unlikely to change this position before the formal vote is undertaken next month.
• In the light of the recent development and the Company's financial situation, the Company will now assess the Company's strategic options and provide an update.
• Price Action: ORPH shares are down 28.7% at $1.44 during the premarket session on the last check Thursday.