Lysosomal storage disorder refers to inherited metabolic disorders that are characterize by enzyme deficiencies, which results in an abnormal build-up of various toxic materials in the body's cells. There are nearly 50 types of lysosomal storage disorder which affect various parts of the body including the brain, skeleton, heart, central nervous system and skin. Some of the lysosomal storage disorders are Aspartylglucosaminuria, Batten Disease, Cystinosis, Fabry Disease, Glycogen Storage Disease II (Pompe Disease), GM2-Gangliosidosis Type I (Tay Sachs Disease), GM2-Gangliosidosis Type II (Sandhoff Disease), Metachromatic Leukodystrophy, Gaucher’s Disease Types I, II, and III, Mucolipidosis Types I, II/III and IV, Mucopolysaccharide Storage Diseases (Hurler Disease and variants, Hunter, Sanfilippo Types A,B,C,D, Morquio Types A and B, Maroteaux-Lamy and Sly diseases), Niemann-Pick Disease Types A/B, C1 and C2 and Schindler Disease Types I and II.
The signs and symptoms vary for different diseases, according to the enzyme deficiency for this group of diseases. Some of the symptoms associated with lysosomal storage disorder include enlargement of the liver (hepatomegaly), rapidly progressive vision failure (optic atrophy), impaired kidney function, burning pain in the hands and feet, chronic fatigue, lethargy, mild mental retardation and abnormalities of the face and skull. Lysosomal storage disorder can be treated by bone marrow transplantation and enzyme replacement therapy (ERT). Some of the marketed drugs for lysosomal storage disorder include alglucerase injection (Ceredase), for treatment of Gaucher’s disease type I; miglustat (Zavesca) for treatment of Gaucher’s disease type I; Fabrazyme, for treatment of Fabry disease and Aldurazyme for treatment for Mucopolysaccharide Type I.
Drug manufacturing companies are constantly involved in the quest to find better and new treatment strategies with potential enzyme replacement therapies such as N-acetylglucosaminidase alpha (NAGLU) replacement which can also prove to be effective in Sanfilippo syndrome type B treatment. Various companies are developing drug candidates using different technologies for the development of effective and better drugs, which is driving the growth of the pipeline.
As of June 2017, the lysosomal storage disorder therapeutics pipeline comprised of approximately 74 drug candidates in different stages of development. The figure below depicts the number of drug candidates and their phases.
LYSOSOMAL STORAGE DISORDER DRUG CANDIDATES UNDER DEVELOPMENT (2017)
Some of the other key players developing drugs for the treatment of lysosome storage disorder include GlaxoSmithKline plc, Sanofi Genzyme, Amicus Therapeutics, Inc. and others.
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