Published: September 10, 2010

Research and Markets: More Than 6,000 Rare Diseases Have Been Identified That Affect a Total of 25 Million People in the United States Alone Says 2010 Report

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DUBLIN - (BUSINESS WIRE) - Research and Markets (http://www.researchandmarkets.com/research/03d33f/dealmaking_and_ind) has announced the addition of Decision Resources, Inc.'s new report "Dealmaking and Industry Outlook in Niche Metabolic Diseases" to their offering.

Biologics will enjoy 12 years of marketing exclusivity under the newly enacted regulatory pathway for biosimilars in the United States, and opportunities for developing and marketing biobetters and enzyme replacement therapies (ERTs; biologics based on therapeutic recombinant enzymes) are great in metabolic disease markets.

In this report, we look beyond the dominant and well-documented diabetes sector of the metabolic marketplace to analyze opportunities in niche metabolic diseases. We chose lysosomal storage diseases because of their unmet need and existing opportunities for developing and marketing more ERTs. We assessed growth-hormonerelated diseases because they represent additional opportunities for developing biosimilars and biobetters.

The report addresses several sectors of the metabolic disease therapy area, the brand drugs used to treat these diseases, and certain R&D pipelines. We also provide insights from interviews with the CEOs of Astellas Venture Management and Takeda Research Investment, and we examine recent dealmaking activities by pharmaceutical companies and certain corporate venture capital firms in metabolism.

Questions Answered in this Report:

More than 6,000 rare diseases (defined as a disease that affects fewer than 200,000 people) have been identified that affect a total of 25 million people in the United States alone. What rare diseases exist in the metabolic disease therapy area? What major brands are marketed to treat some of these diseases? What treatment options are in development for these diseases? What manufacturing problems has Genzyme experienced with its enzyme replacement therapies?
Human growth hormones were among the earliest biosimilars on the market. Why is Novartis more strongly committed to its generics subsidiary Sandoz today than in the past? What are the major marketed growth hormone products? What is the status of growth hormone biosimilars and biobetters? Why will the human growth hormone biosimilar market be slow to develop?
GlaxoSmithKlines CEO Andrew Witty told investors that the cost of achieving success in the company's neuroscience programs was disproportionately high. Why did GlaxoSmithKline disinvest in its neuroscience programs for pain, anxiety, and depression? Why did GlaxoSmithKline establish a new rare disease unit? What recent deals form the basis of this new endeavor? Why does GlaxoSmithKline feel that the risk associated with product discovery and development in rare diseases is generally lower than in other disease areas?
Astellas Pharmas senior director for licensing and alliances, Yoshitaka Yoneyama, says that Astellas Venture Management (AVM) promotes direct dialogue between portfolio companies and company headquarters. What are the strategies of corporate venture capital (CVC) groups? Why do CVC groups dislike pay to play deals? In which metabolism companies have AVM and Takeda Research Investment invested? What perceptions do CVCs have about metabolic disease drug discovery and development?
Teva North America CEO Bill Marth says that his company never saw the new U.S. biosimilars pathway as the only way to compete when biologics lose patent protection and that Teva is going the biologics license application (BLA) route for many biologics. Why does Teva feel that the U.S. Congress dropped the ball on biosimilars legislation? Why is Teva planning to sidestep the new U.S. biosimilars pathway? Why is drug development of orphan diseases such as lysosomal storage diseases uniquely suited to take advantage of the 12-year market exclusivity afforded to original biologics in the United States?

Scope:

Primary research: Interviews with the CEO of Astellas Venture Management, the CEO of Takeda Research Investment, and Astellas Phamas senior director for licensing and alliances. Decision Resources analysts market forecasts of therapies for metabolic diseases, lysosomal storage diseases, and growth-hormone-related diseases.
Diseases: Metabolic disease, lysosomal storage disorders, mucopolysaccharidoses, Hurler-Scheie disorder, Hunters syndrome, Morquios syndrome, Sanfi lippos syndrome, Slys syndrome, Maroteaux-Lamy syndrome, sphingolipidoses, metachromatic leukodystrophy, Tay-Sachs disease, Krabbes disease, Wolmans disease, cholesteryl ester storage disease, Gauchers disease, Fabrys disease, Niemann-Pick disease, glycogen storage disorders, Pompes disease, von Gierkes disease, Andersens disease, McArdles disease, Coris disease, Hers disease, Taruis disease, Fanconi-
Bickels syndrome, mucolipidoses, cherry-red-spot-myoclonus syndrome, inclusion-cell disease, growth-hormone deficiency, idiopathic short stature, small for gestational age (SGA), acromegaly, AIDS-associated wasting and cachexia, growth failure from chronic renal insuffi ciency (CRI), Noonans syndrome, Prader-Willis syndrome, short stature homeobox (SHOX) defi ciency, Turners syndrome, obesity, type 1 and type 2 diabetes.
Therapies in development: Enzyme replacement therapy (ERT); substrate reduction therapy (SRT); RNA-targeting drugs; stop codon read through therapy; gene replacement therapy; pharmacological chaperone therapy (PCT); hematopoietic stem cell transplantation (HSCT); enzyme enhancement therapy (EET); gene mutations; disease-causing enzymes; major brands for lysosomal storage disorders, type 1 and type 2 diabetes, and obesity; R&D pipelines for mucopolysaccharidoses, sphingolipidoses, and glycogen storage disorders; international classifi cation codes for endocrine, nutritional, and metabolic diseases; Genzymes ERT manufacturing problems, gastrointestinal permeation enhancement technology (GIPET), xenotransplantation, cell regeneration.
Dealmaking and strategies: Recent metabolic disease deals for 13 companies, new focus on rare diseases, corporate venture capital strategies and deals, biologics strategies, biosimilars, biobetters, biologic market exclusivity opportunities, premium pricing of orphan disease therapies.

Special features of this report include:

Interviews with the CEOs of Astellas Venture Management and Takeda Research Investment
New rare disease strategies by Big Pharma.
Deficient genes and defi cient enzymes for lysosomal storage disorders.
R&D pipelines for mucopolysaccharidoses, sphingolipidoses, and glycogen storage disorders.
Major brands for lysosomal storage disorders, type 1 and type 2 diabetes, and obesity.
An appendix of international classifi cation codes for endocrine, nutritional, and metabolic diseases.

Key Terms for this Report: Metabolic, lysosomal storage, mucopolysaccharidosis, sphingolipidosis, glycogen storage, mucolipidosis, growth hormone, corporate venture capital, inlicensing, biologic, biosimilar, market exclusivity, premium pricing, enzyme replacement therapy, substrate reduction therapy.

Key Topics Covered: