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Our VISION

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

 
 

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.

 

News

Featured
ORPHAZYME CMO TO SPEAK AT FDA-FUNDED RARE DISEASE DATA ANALYTICS PLATFORM LAUNCH EVENT

Copenhagen, Denmark, September 17, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, has been invited to participate in a meeting hosted by the National Organization for Rare Disorders (NORD) and the Critical Path Institute (C-PATH) to launch their joint initiative: the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP). Funded by the US Food and Drug Administration (FDA), the goal of RDCA-DAP is to accelerate the process of moving therapies from bench to bedside for rare diseases. 

 
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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.

 
 

NPC Trial

Learn more about our trials in NPC.

Social Media

Are you a patient, caregiver or healthcare professional? Do you need more information about our approach? We want to hear from you!  Contact us at contact@orphazyme.com.

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