Enzyme replacement therapy involves intravenous administration of enzymes in order to correct the deficiency of enzymes that causes a specific disease condition called lysosomal storage disease. Enzyme replacement therapy is used in Fabry Disease, Gaucher disease, lysosomal acid lipase deficiency, mucopolysaccharidosis, Hunter syndrome, and Pompe disease. Enzymes are obtained from sources such as human cells, animal cells, and recombinant DNA technology. Enzyme replacement, although does not provide permanent cure, helps prevents permanent damage to the body caused due to deficiency of a specific enzyme.

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Treatment consists of weekly or monthly doses depending upon the disease. For instance, in order to address enzyme deficiency in Gaucher disease, modified version of glucocerebrosidase is administered intravenously every two weeks. Enzyme replacement is associated with fewer side effects than other treatment methods. However, enzyme replacement therapy may be inconvenient for some patients such as children and geriatric population due to intravenous administration. It may result in local infusion reaction or hypersensitive reactions in the form of local rash, fever, edema, bronchospasm or hypotension.

Increasing collaborations amongst key companies to fast-track the development of enzymes for replacement is expected to boost growth of the enzyme replacement therapy market size. For instance, in March 2018, Shire plc entered into a preclinical research collaboration with NanoMedSyn— French biotech company—to develop enzyme replacement treatment for a lysosomal storage disorder based on NanoMedSyn’s synthetic derivative technology named AMFA.

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Key Vendors:

Shire plc, Amicus Therapeutics, Genzyme Corporation, Pfizer Inc., BioMarin Pharmaceutical, Inc., Sigma-Tau Pharmaceuticals, Inc., Essential Pharmaceuticals Limited, Merck KGa, and AbbVie Inc.

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