Pioneering a new kind of treatment

Orphazyme is pioneering the development of a new type of medical treatment by applying our specialized expertise in heat shock proteins—a key component of the body’s natural cellular stress-defense system.

By leveraging the potential of heat shock proteins, such as heat shock protein 70 (HSP70), we are advancing treatments for patients living with a range of neurodegenerative orphan diseases. These diseases can have a devastating impact, and the lack of existing approved treatment options leaves a substantial gap in care today.




Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C

Orphazyme A/S Company announcement No. 16/2021 Inside information Company Registration No. 32266355 Copenhagen – June 18 , 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today