Who we are

Orphazyme is a Danish biopharmaceutical company with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases.

We develop new therapies that mobilize a group of stress-response proteins, called heat-shock proteins. Heat-shock proteins help restore balance to cells affected by stress arising from misfolded proteins, protein aggregates, and lysosomal dysfunction.

Our pipeline comprises three potential registration studies, with the first potential marketing authorization expected in 2020.

Disease focus

Protein-aggregation diseases
Protein aggregation may occur as a consequence of protein misfolding and underlies a large number of diseases, including neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease. Other less prevalent protein-aggregation diseases include amyloidosis, Amyotrophic Lateral Sclerosis (ALS), and the muscle-wasting disease sporadic Inclusion Body Myositis (sIBM). Orphazyme currently conducts a clinical trial for the protein-aggregation disease sIBM and are planning a clinical trial for ALS.

Lysosomal storage diseases
Lysosomal storage diseases are inherited metabolic disorders where enzyme deficiencies result in an accumulation of toxic materials in the cells of the body. These deficiencies are often caused by mutations leading to premature misfolding and degradation of the enzymes. 
Among the group of lysosomal storage diseases, Orphazyme is currently conducting clinical trials for NPC and planning a clinical trial for Gaucher disease.

We are currently investigating the safety and efficacy of arimoclomol in Niemann-Pick disease Type C (NPC). NPC is a rare, lysosomal storage disorder which affects around 1 in 120,000 newborns.