Phase II Trial for Investigational Gaucher Disease Drug Begins

According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced the dosing of its first patient in its Phase II clinical trial.

The experimental drug to best tested in the trial is arimoclomol; in this trial, its effectiveness will be tested against Gaucher disease.

Gaucher disease is a type of genetic disorder that is characterized by the build up of glucocerebroside within cells and certain organs. It caused be a deficiency of an enzyme that, under typical circumstances, regulates the concentration of glucocerebroside in the body. Gaucher disease causes a number of symptoms, such as enlargement of the liver and spleen, the premature death of blood cells (multiple types), joint and bone pain, osteoporosis, yellow-brown skin color, and neurological symptoms (convulsions, intellectual disability, dementia) that only occur in certain variants of the illness. The standard of care for Gaucher disease is enzyme replacement therapy to supplement the deficient enzyme. Gaucher disease is the most common of the lysosomal storage diseases. There are an estimated 10,000-15,000 patients in Europe and the US. To learn more about Gaucher disease, click here.

Arimoclomol is meant to increase the production of heat shock proteins. These proteins can help reduce the problems caused by misfolded proteins and improve the functionality of lysosomes. Arimoclomol can cross the blood-brain barrier and can be administered orally. It is currently in development for treating a variety of different rare diseases, including amyotrophic lateral sclerosis, Niemann-Pick disease Type C, and sporadic inclusion body myositis. The drug is currently Orphazyme’s leading therapeutic candidate.

The trial will take place at multiple clinical sites located in India and will include 40 patients. The patients will receive a placebo or arimoclomol for a period of six months. The results of the clinical trial are expected to be released early next year.

Hopefully, arimoclomol will demonstrate its effectiveness in the Phase II trial, as treatments for Gaucher disease are limited, and new options for patients are desperately needed.


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