Recombinant enzymes are indicated for a family of conditions called lysosomal storage disease. In a lysosomal storage disorder, certain structures in cells, called lysosomes, have trouble metabolizing (breaking down) specific complex fatty molecules. The lysosomes fill up with these undigested molecules, impairing the cell’s ability to function properly. The recombinant enzymes covered in detail in this report are administered as a form of enzyme replacement therapy. Because they afflict a relatively small segment of the population, Lysosomal storage disorders are classified as orphan diseases. The combination of high manufacturing costs associated with host system manufacturing and relatively low quantities result in high cost-per-prescription prices. Cerezyme was the first drug in this class to receive marketing authorization in 1994. Slightly more than half of recombinant enzymes are sourced in the US, with the remainder manufactured in Europe. This report examines the current market and the probable developments for the remainder of the decade.
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What You Will Learn
• What are the noninvasive recombinant enzyme products that are have been given marketing authorization by the FDA or the EMA?
• Who supplies the API; the finished product? Who is the labeler; the marketer? What is the host system?
• Who are the major recombinant enzyme suppliers; what are their facilities, and where are they located?
• What are the as-supplied characteristics for each enzyme product and how are they administered?
• What is the probable impact of biosimilars on approved enzyme products in North America; in Europe?
• What does the market look like today? What is the forecast for how it will look in 2022?
• What will be the impact of economic, technology, and regulatory factors on the commercial potential of recombinant enzymes?
Recombinant Enzymes – Summary of Contents
Recombinant Enzymes – The Market Opportunity
Treating Hereditary Diseases
Therapeutic Demand Drivers
Sourcing Recombinant Enzymes
Collaborations & Relationships
Recombinant Enzymes – Indications & Markets
Lysosomal Acid Lipase Deficiency
Mucopolysaccharidosis Type IVA
Mucopolysaccharidosis Type VI
Type 1 Gaucher Disease
Recombinant Enzymes – Product Analysis
Recombinant Enzymes – Market Data & Forecasts
Packaging and Administration
Managed Care Initiatives