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.
The company was based on a scientific discovery on the function of Heat-Shock Proteins (HSPs) by Thomas Kirkegaard Jensen and Professor Marja Jäättelä that was published in the scientific journal Nature. Since inception, Orphazyme has translated the scientific discovery into a late-stage clinical development program.
To profoundly impact the lives of patients with orphan diseases and their families.
Bringing innovative therapies to patients with rare, serious diseases.
The strategy is to develop treatments for orphan diseases with protein misfolding where it can apply its specialized know-how in HSPs.
The objective is to successfully conduct and complete the planned and on-going trials of arimoclomol for the treatment of protein-aggregation diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and the lysosomal storage diseases, Niemann Pick disease Type C (NPC) and Gaucher disease.
As the clinical development program for arimoclomol progresses, Orphazyme intends to refine and finalize its commercialization strategy and build its commercial structure and operations. The company intends to build its own sales force in key markets. In markets outside the USA 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, hence the development of a proprietary suite of New Molecular Entities (NMEs) with improved characteristics. Orphazyme intends to select diseases suitable for the NMEs based on genetic and mechanistic insights into selected protein-misfolding diseases.
Care for people with rare diseases with a high unmet medical 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.
Chief Executive Officer
Chief Medical Officer
Chief Scientific Officer
Chief Commercial Officer
Chief Financial Officer
Chairman of the board
Deputy Chairman of the board