Some 36 orphan drugs
have been approved by the FDA under the Orphan Drug
Act and are available to patients.
Prescription Orphan Drug
and Biologic Developers and Marketers
present opportunities to pharma companies looking to move into more niche indications and away from mass market indications that have very low levels of clinical unmet need.
Fully updated for 2010, with complete lists of orphan drugs
approved in the US, Japan and Korea.
The main emphasis though is not on registration but on market access, which for products like orphan drugs
that often carry premium prices, means eligibility for reimbursement or other sources of public funds.
Assesses the future size and growth of the orphan drugs
The orphan drug
market is broken down into applications of biologics and non-biologics.
The contrasting requirements and different time frames in which this legislation has been enacted have also resulted in considerable differences in the numbers of designated and approved orphan drugs
within each market.
As of October 2002, 74 orphan drugs
had achieved market approval status by virtue of the orphan drug
NEW YORK -- Since its passage in 1983, the Orphan Drug
Act--legislation to facilitate development of orphan drugs
, defined as medications for rare conditions such as Huntington's Disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy --has long been considered a success for encouraging the production of hundreds of medications for the Americans suffering from these diseases.
The court found that the Act's plain language generally prohibits the FDA from approving orphan drugs
for the same indications that have previously been approved and designated.
are either drugs or biologics intended for the treatment, diagnosis or prevention of rare diseases which are defined as life-threatening or chronically debilitating conditions affecting less than five in 10,000 people in the European Union per year.