Orphazyme was founded in 2009 with the objective to develop new therapies for patients suffering from protein misfolding diseases with no or limited treatment options available.

At inception, the Company was based on a scientific discovery on the function of HSPs by Thomas Kirkegaard Jensen (current CSO) and Professor Marja Jäättelä that was published in the scientific journal Nature. Since inception, the Company has translated the scientific discovery into a late stage clinical development programme.




To profoundly impact the lives of patients with orphan diseases and their families.




The strategy is to develop treatments for orphan diseases with protein misfolding where it can apply its specialised know-how in HSPs.   

The objective is to successfully conduct and complete the planned and ongoing trials of arimoclomol for the treatment of the neuromuscular diseases, sIBM and ALS, and the lysosomal storage diseases, NPC and Gaucher disease. 

As the clinical development programme for arimoclomol progresses, Orphazyme intends to refine and finalise its commercialisation strategy and build its commercial structure and operations. The Company intends to build its own sales force in key markets. In markets outside the United States and Europe, Orphazyme currently intends to partner with local/regional distributors or license partners in certain other geographical areas. 

Protein misfolding is the hallmark of a broad range of diseases and Orphazyme’s strategy is to use its expertise, including proprietary know-how to select and develop new leads for suitable diseases. In line with this, the Company is developing a proprietary suite of NMEs with improved characteristics. Orphazyme intends to select diseases suitable for the NMEs based on genetic and mechanistic insights into selected protein misfolding diseases. 



Core values

Care for people with rare diseases, and with diseases of a high unmet need.

Work tirelessly to conduct the most thorough, diligent research and bring results to the lives of patients as quickly and safely as possible.

Build powerful relationships with everyone connected to the diseases we are treating, to learn from the wider community.