Orphazyme withdraws MAA for arimoclomol

Shares of Danish biopharma firm Orphazyme (CPH: ORPHA) were down 4.8% at 5.90 kroner by late morning today, after it announced a setback with the regulatory filing for its lead product candidate in Europe.

Following the receipt of the negative Trend Vote by the Committee for Medicinal Products for Human Use (CHMP) as announced on February 23, 2022, Orphazyme says it has decided to withdraw its European Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC) ahead of a final vote and opinion by the European Medicines Agency’s CHMP on the MAA scheduled for later this month.

“Today’s decision to withdraw the MAA enables us to consider the best possible path forward for arimoclomol,” commented Orphazyme chief executive Anders Vadsholt, adding: “NPC is an ultra-rare neurodegenerative disease with a high unmet medical need, and we want to explore options for arimoclomol in this indication, to deliver on our commitment to patients with this devastating disease.”