 DR Reports --
July 2005
 In This Issue...
Introduction
Orphan drugs, which are developed for the treatment of rare (orphan) diseases,
have recently seen increased commercial success. The creation of orphan
drug legislation between 1983 and 2000 in the United States, the European
Union, and Japan has clearly encouraged the development of new treatments
for rare and relatively rare diseases.
This report examines the pipeline of drugs in development under the guidance
of orphan drug legislation in major markets (United States, Europe, and
Japan). Each of these markets has orphan drug legislation with different
provisions, and we compare these regulatory factors. We also profile companies
that are active in the development of orphan drugs. Finally, we examine
how the orphan drug market is likely to evolve over the next ten years
by considering the influence that such factors as patent issues, pharmacogenomics,
and reimbursement will have on company strategy.
Key Players
The eight companies whose primary strategy is the development of orphan
drugs focus their activities on their domestic markets, but many have developed
alliances with one another as well as with other companies not focused
on the orphan drug market. Orphan Medical and Rare Therapeutics are the
only two such companies in the United States; four companies--Orphan Pharma
International, Orphan Europe, AOP Orphan Pharmaceuticals, and Swedish Orphan--are
based in Europe. Orphan Link is the only such company based in Japan, and
Orphan Australia is based in Australia.
A number of emerging players within the orphan drug market are focused
on the development of large-molecule therapeutics. Genzyme is the most
established of these companies and is focused on the development of enzyme
replacement therapies for certain genetic diseases. Other emerging companies
are Transkaryotic Therapeutics, Sigma Tau, Amicus Therapeutics, Ark Therapeutics,
BioMarin Pharmaceutical, Seattle Genetics, and ZyStor Therapeutics.
Why Buy This Report?
- Learn what the leading players in the orphan drug market are doing via
detailed analyses of their pipelines.
- Gain valuable insight regarding emerging companies and the agents in their
orphan drug pipelines.
- Explore key trends in the orphan drug market and their potential
impact on the commercial outlook for this market.
- Assess marketed and pipeline drugs with respect to development
phase and peak-year sales potential.
- Analyze orphan drug alliance activity presented in comprehensive
company profiles and evaluate company financial information.
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