About Enzyme Replacement Therapy
A rare disease is defined as a disease that affects a small percentage of the general population. Drugs used for the treatment of rare diseases are called orphan drugs. Enzyme Replacement Therapy (ERT) is the treatment for rare diseases. According Global genes.org, 80% of rare diseases are of genetic origin. There are approximately 7,000 different types of rare diseases. Approximately 50% of the people affected by rare diseases are children. According to the Kakkis Every life Foundations, FDA-approved drugs are not available for 95% of rare diseases. ERT replaces an enzyme that is absent in the body. ERT is available for diseases such as fabry disease, gaucher disease, mucopolysaccharidosis, Pompe disease, and lysosomal acid lipase deficiency, among others.
Attributes | Details |
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Study Period | 2018-2030 |
Base Year | 2023 |
Unit | Value (USD Million) |
The companies are exploring the market by adopting expansions, investments, new service launches, and collaborations as their preferred strategies. The players are exploring new geographies through expansions and acquisitions across the globe to gain a competitive advantage through combined collaborations. Analyst at AMA Research estimates that United States Players will contribute the maximum growth to Global Enzyme Replacement Therapy market throughout the forecasted period. Established and emerging Players should take a closer view at their existing organizations and reinvent traditional business and operating models to adapt to the future.
Johnson & Johnson (United States), Sanofi (France), Shire (United States), BioMarin (United States), AbbVie (United States), Alexion (United States), Allergan (Ireland), Horizon Pharma (Dublin), Actelion (Switzerland) and Recordati Rare Diseases (United States) are some of the key players that are part of study coverage. Additionally, the Players which are also part of the research coverage are Digestive Care and Leadiant Biosciences.
Segmentation Overview
AMA Research has segmented the market of Global Enzyme Replacement Therapy market by , Application (Gaucher Disease, Fabry Disease, MPS Disease, Gastrointestinal Disease and Others) and Region.
On the basis of geography, the market of Enzyme Replacement Therapy has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). If we see Market by Enzyme , the sub-segment i.e. Imiglucerase will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth. If we see Market by Route of Administration, the sub-segment i.e. Injectable Agents will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth. If we see Market by Distribution Channel, the sub-segment i.e. Hospital Pharmacies will boost the Enzyme Replacement Therapy market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
Influencing Trend:
Advancement in the medical sector
Market Growth Drivers:
Increase in healthcare expenditure and prevalence of rare diseases and Growth in the demand for ERT to improve the treatment of rare diseases
Challenges:
lack of regulatory framework
Restraints:
Availability of alternative treatment options and Dearth of skilled healthcare professionals
Opportunities:
Increase in government spending on healthcare infrastructure, such as favorable reimbursement for ERT
Market Leaders and their expansionary development strategies
In November 2020, Retrophin, Inc. announced the completion of its previously announced acquisition of Orphan Technologies Limited, a privately held, clinical-stage biopharmaceutical company focused on the development of product candidate OT-58 for the treatment of classical homocystinuria (HCU). OT-58 is a novel investigational human enzyme replacement therapy being evaluated in Phase 1/2 development for the treatment of classical HCU, a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to life-threatening thrombotic events such as stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delay.
In November 2023, Takeda launched Adzynma (ADAMTS13, recombinant-krhn) to treat adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). The FDA granted approval to Adzynma in early November 2023 as a prophylactic or on-demand enzyme replacement therapy.
Key Target Audience
New Entrants/Investors, Analysts and Strategic Business Planners, Enzyme Replacement Therapy Provider, Government Regulatory and Research Organizations and End-Use Industry
About Approach
To evaluate and validate the market size various sources including primary and secondary analysis is utilized. AMA Research follows regulatory standards such as NAICS/SIC/ICB/TRCB, to have a better understanding of the market. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with the purpose to understand the companies positioning regarding the market value, volume, and their market share for regional as well as global.
Further to bring relevance specific to any niche market we set and apply a number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Agencies) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders, etc., and work towards appointment generation.
The primary research is performed by taking the interviews of executives of various companies dealing in the market as well as using the survey reports, research institute, and latest research reports. Meanwhile, the analyst team keeps preparing a set of questionnaires, and after getting the appointee list; the target audience is then tapped and segregated with various mediums and channels that are feasible for making connections that including email communication, telephonic, skype, LinkedIn Group & InMail, Community Forums, Community Forums, open Survey, SurveyMonkey, etc.