Competitor Analysis: Enzyme Replacement Therapies

The Competitive Intelligence Report Enzyme Replacement & Enhancement Therapies (ERT) as of December 2008 provides a competitor analysis in the development pipeline of novel enzyme replacement therapies and of enzyme enhancement therapies to treat human genetic diseases and exocrine pancreatic insufficiency due to cystic fibrosis, pancreatitis and other causes. The report includes an evaluation of the 2007 sales of the major products based on published data and provides company product portfolios and R&D pipelines in the field of ERT.

Recombinant DNA technology strongly has facilitated the supply of high quality products for treatment of human genetic diseases by enzyme replacement. Rare genetic diseases such as Gaucher disease, Fabry disease, Pompé disease , Hunter disease and other lysosomal storage diseases now effectively can be treated by replacement of the deficient enzymes with recombinant proteins.  2007 sales of the recombinant enzyme replacement therapies (ERT) reached a record level of US$ 2.3 bln shared predominantly by three companies (Genzyme, Shire and Biomarin Pharmaceuticals) for a total of seven different products. Companies are working to extending the market by developing novel ERTs for further human genetic diseases as well as by profiling small molecule therapies for enhancement of enzymeti cactivities. Pharmacologic chaperons serve to bind and stabilize enzyme activities and have the advantage of oral administration. Other next generation therapies include long-acting enzymes or those produced by low-cost or alternative technologies to circumvent patent exclusivity.

Porcine-derived extracts containing pancrelipase (a mixture of lipase, amylase and protease among others) recently were forced by the FDA to undergo regulatory review of an NDA. Now these products are exposed to upcoming competition with enzymes produced by recombinant DNA technology which intent to capture a part or maintain existing sales of exocrine enzyme replacement therapies (2007 sales > US$ 300 mln).

The report includes a compilation of current active projects in research and development of novel enzyme replacement & enhancement therapy products and an overview of approved products. Competitor projects are listed in a tabular format providing Information on:

• Drug Codes,
• Target / Mechanism of Action,
• Class of Compound,
• Company,
• Product Category,
• Indication,
• R&D Stage and
• additional comments with a hyperlink leading to the source of information.