Who we are
Orphazyme is a Danish biotech company with a late stage drug pipeline, developing new treatment options for orphan protein misfolding diseases.
The Company’s lead candidate, arimoclomol, stimulates the production of the cells’ protein rescue system, the heat shock proteins (“HSPs”), which helps the misfolded proteins to either get back to their functional shape or, if this cannot be achieved, removing them from the cells by means of the cells’
recycling system, the lysosomes, so they no longer form toxic aggregates.
Currently, the Company expects to have completed three potential registration studies by the end of 2020 with the first potential marketing authorisation expected in 2020.
Neuromuscular disorders encompass a range of conditions affecting a part of the neuromuscular system and impairing the functioning of the muscles, thereby causing muscle weakness and fatigue. Among the group of neuromuscular disorders, Orphazyme currently conducts a clinical trial for the diseases sIBM and 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 type C (NPC). NPC is a rare, lysosomal storage disorder which affects around 1 in 150,000 newborns.
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