Enzyme replacement therapy is a medical treatment, which involves replacement of enzymes that are absent of inadequate in the body.
Seattle, WA -- (SBWIRE) -- 09/25/2019 -- Global Enzyme Replacement Therapy Market, by Enzyme Type (Agalsidase Beta, Velaglucerase Alfa, Imiglucerase, Taliglucerase, Alglucosidase, Alfa, Laronidase, Idursulfase, Galsulfase, Pegademase, and Others), by Indication (Fabry Disease, Gaucher Disease, Pompe Disease, Scheie Syndrome, Hunter Disease, Maroteaux-Lamy Syndrome, and Others), by End User ( Hospitals, Clinics, and Others), and by Geography - Size, Share, Outlook, and Opportunity Analysis, 2018-2026
Enzyme replacement therapy is a medical treatment, which involves replacement of enzymes that are absent of inadequate in the body. The therapy involves intravenous administration of enzymes to correct the deficiency of enzymes. This deficiency is caused due to a typical disease called lysosomal storage disease. This therapy also finds it application in Gaucher disease, Fabry disease, Pompe disease, Hunter syndrome, lysosomal acid lipase deficiency, and mucopolysaccharidosis. Enzymes used for replaced are obtained from human cells, animal cells, and recombinant DNA technology. Although enzyme replace does not provide permanent cure, it helps to prevent perpetual damage to body caused due to deficiency of a specific enzyme. Its treatment includes doses that can be either weekly or monthly, depending upon the disease. For instance, in Gaucher disease, improved version of glucocerebrosidase is administered intravenously every two weeks, in order to tackle the enzyme deficiency. One of the major advantages enzyme replacement therapy provides is that it has fewer side effects than other contemporary methods. However, enzyme therapy may not be convenient for some patients, typically geriatric patients and children due to its mode of administration. It could result in local infusion reaction and hypersensitive reactions in the form of fever, edema, rash, bronchospasm or hypotension.
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Market Dynamics
Even though lysosomal storage disease is an odd disorder, there are 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. It consists of 60 genetic abnormalities with problematic enzyme function. As this is an orphan disease condition, regulatory authorities tend to offer the benefit of rapid approval and ease in approval process. This has led to introduction of new therapies by key players, which is expected to drive growth of the global enzyme replacement therapy market. For instance, in November 2017, The U.S. Food and Drug Administration (FDA) approved Mepsevii (vestronidase alfa), which marked as the first authorized treatment for pediatric and adult patients suffering from an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII). Mepsevii received the fast-track approval and orphan drug status from the U.S. FDA to incentivize the development. Moreover, financial help by NGOs such as National Gaucher Society is expected to help patient receive enzyme replacement therapy that are expensive.
Furthermore, increasing collaborations activities among key players in the market to fast-track the development of enzymes for replacement is expected to boost the global enzyme replacement therapy market growth. For instance, in March 2018, Shire plc, a biopharmaceutical company, entered into a preclinical research collaboration with NanoMedSyn, a 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.
Regional Insights
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.
Competitive Landscape
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).
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Market Taxonomy
On the basis of enzyme type, the global enzyme replacement therapy market is segmented into -:
- Agalsidase Beta
- Velaglucerase Alfa
- Imiglucerase
- Taliglucerase
- Alglucosidase Alfa
- Laronidase
- Idursulfase
- Galsulfase
- Pegademase
- Others
On the basis of indication, the global enzyme replacement therapy market is segmented into -:
- Fabry Disease
- Gaucher Disease
- Pompe Disease
- Scheie Syndrome
- Hunter Disease
- Maroteaux-Lamy Syndrome
- Others
On the basis of end user, the global enzyme replacement therapy market is segmented into -:
- Hospitals
- Clinics
- Home care setting
On the basis of geography, the global enzyme replacement therapy market is segmented into -:
- North America
- Latin America
- Europe
- Asia Pacific
- Middle East
- Africa
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