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. 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.
Although lysosomal storage disease is a rare disorder, a significant number of people are affected with this disease. According to study published in Molecular Genetics and Metabolism Reports in December 2017, lysosomal storage disease has incidence rate of 1 in 4000 to 1 13,000 live births. Lysosomal storage disease consists of 60 genetic abnormalities with problematic enzyme function. Being an orphan disease condition, regulatory agencies offer the benefit of rapid approval and ease in approval process. This in turn, is expected to expand the overall enzyme replacement therapy market with introduction of new therapies by various key players. For instance, in November 2017, The FDA approved Mepsevii (vestronidase alfa), which is first authorized treatment for pediatric and adult patients suffering from an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII). FDA granted Mepsevii with fast-track approval and orphan drug status to incentivize the development. Financial help by NGOs such as National Gaucher Society help patient receive enzyme replacement therapy that are costly.
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.
However, high cost associated with the enzyme replacement therapy and complex reimbursement scenario causing therapy to be excluded from coverage by insurers due to this being a rare disease, hamper growth of the enzyme replacement therapy market.
On the basis of geography, the global enzyme replacement therapy market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America is expected to lead the market during forecast period, followed by Europe. According to study published in journal Hematology, in March 2017, incidence of Gaucher Disease in the general population varied from 0.39 to 5.80 per 100?000, and its prevalence varied from 0.70 to 1.75 per 100?000. Ease in access and government support for research and development is expected to aid growth of the enzyme replacement therapy market.
Key players operating in the global enzyme replacement therapy market include Shire plc, Amicus Therapeutics, Genzyme Corporation, Pfizer Inc., BioMarin Pharmaceutical, Inc., Sigma-Tau Pharmaceuticals, Inc., Essential Pharmaceuticals Limited, Merck KGa, and AbbVie Inc.
Companies are undertaking significant amount of research for development of new enzyme replacement therapies, in turn being benefitted from orphan drug status and fast-track approval. Amicus Therapeutics was granted with orphan drug designation for its investigational combination drug therapy ATB200/AT2221 in November 2017. This drug is intended to cure Pompe disease (acid maltase deficiency or AMD).
On the basis of enzyme type, the global enzyme replacement therapy market is segmented into -:
On the basis of indication, the global enzyme replacement therapy market is segmented into -:
On the basis of end user, the global enzyme replacement therapy market is segmented into -:
On the basis of geography, the global enzyme replacement therapy market is segmented into:
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