Clinical Trials for Krabbe disease

Find the latest information related to clinical trials for Krabbe disease in this category.


On 27 September 2011, the European Commission has designated the drug "recombinant human galactocerebrosidase" (Galaczym) of ACE BioSciences A / S, a subsidiary of Zymenex Holding A / S as an orphan drug for the indication: Treatment of KrabbeCe disease drug is entered in the Community Register of Orphan Medicinal Products under number EU/3/11/911.

On 12 December 2011, Galaczym also obtained orphan drug designation by the U.S. Food and Drug Administration (FDA), Health Authority of North America.

Orphan designation is an initiative to encourage pharmaceutical companies to develop treatments for rare diseases. It is accompanied by incentives to promote research, development and
market designated medicinal product. It is in no way authorized therapeutic trial or marketing.

Krabbe disease (also known as globoid cell leukodystrophy) is an inherited disease due to lack of an enzyme called galactocerebrosidase is necessary for the degradation of certain fats such as galactosylceramide and psychosine.
The accumulation of these substances is responsible for the destruction of the myelin cells to cause lesions in the brain.