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早く治療薬の開発が煤も事を祈ります。
Zymenex enzyme gets US Orphan Drug Designation Approval from FDA
The US Food and Drug Administration (FDA) has, on December 12, 2011 granted the Danish biotech company ACE BioSciences A/S (a subsidiary of Zymenex Holding A/S) Orphan Drug Designation for its recombinant human enzyme Galaczym, for the treatment of the rare, lysosomal disease Globoid Cell Leukodystrophy (Krabbe Disease). Orphan designation qualifies the sponsor of the product for fee reductions, protocol assistance and access to fast track approval and 7 years marketing exclusivity once the product has been approved for the market, according to the US Orphan Drug Act. Zymenex has already received Orphan Drug Designation in the EU for Galaczym in September 2011 and has previously received Orphan Drug Designation in both the EU and US for two other lysosomal enzyme products, Metazym for the treatment of Metachromatic Leukodystrophy and Lamazym for alpha-Mannosidosis, which has recently, successfully completed Phase 2a clinical trials.
Globoid cell leukodystrophy (also known as Krabbe disease) is a hereditary disease that is caused by the lack of an enzyme (a specialised type of protein) called galactocerebrosidase (GALC). This enzyme is needed to break down certain fatty substances including two lipid substances called galactosylceramide and psychosine. The accumulation of these substances is thought to destroy the cells that produce myelin, the protective sheath that surrounds the nerve cells, resulting in nerve damage in the brain and other parts of the body. The symptoms include extreme irritability or hypersensitivity, spasticity, and developmental delay. The symptoms and signs of central nervous system involvement are severe and intellectual deterioration is seen, leading to lack of voluntary movement and contact with the surroundings. Krabbe Disease is a long-term debilitating disease that can be life-threatening particularly in the early onset form, which typically leads to death in early infancy.
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