Orphan drug


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orphan drug

[ôr′fən]
Etymology: Gk, orphanos, without parents; ME, drogge
a term that generally refers to drugs needed to treat rare diseases but can encompass any pharmaceutic product available to physicians and patients in countries other than the United States that has not been "adopted" by a domestic pharmaceutic manufacturer or distributor. An orphan drug may not be available in the United States because total sales would not justify the expense of research and development or because the medication may be a natural substance that cannot be effectively protected by patent laws against competition from a similar form of the product. The U.S. Orphan Drug Act of 1983 offers federal financial incentives to commercial and nonprofit organizations to develop and market drugs previously unavailable in the United States for rare diseases affecting fewer than 200,000 people. The FDA assists in the process with its office of Orphan Product Development.
Any drug, biological, medical device, or food of potential or actual use in treating ‘orphan’ diseases—diseases regarded by the pharmaceutical industry as too rare for developing commercially viable products

orphan drug/product

Any drug, biological, medical device, or food of potential or actual use in treating 'orphan' diseases–diseases regarded by the pharmaceutical industry as too rare for developing commercially viable products. See Orphan disease. Cf Pseudoorphan drug.

Orphan drug

A term for a drug that treats a rare disease, defined by the Food and Drug Administration (FDA) as one that affects fewer than 200,000 Americans. The FDA has an Office of Orphan Products Development (OOPD), which offers grants to researchers to develop these products.
References in periodicals archive ?
One example of the financial implications of the Orphan Drug Act is Celgene, which has been one of the top beneficiaries of the legislation.
Until Depomed, orphan drug exclusivity was automatic when the statutory criteria were met and if the FDA had not already approved and designated an orphan drug for the same use or indication.
Understand market trends: Identify the top 20 brands with FDA orphan drug approval and their forecast values for 2017.
The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics.
Orphan drug designation qualifies the product for seven years of market exclusivity in the US upon FDA approval of a new drug application for the orphan designated indications.
This review concludes that two of every three orphan drugs approved show the FDA's flexibility in its review of effectiveness data on orphan drug therapies," says Sasinowski.
According to the company, this Orphan Drug Designation complements the corresponding designation in the US from FDA, granted in August 2013.
based biotechnology company (through its Ceptor unit) has applied for orphan drug status for Myodur, a drug for muscular dystrophy.
In 1983, Congress passed the Orphan Drug Act to encourage development of pharmaceuticals used in the treatment or diagnosis of diseases that occur so rarely that there is no financial incentive for development of these so-called "orphan drugs.
Market conditions: provides an analysis of the orphan drug market, detailing the drivers for recent growth, prevalence and scientific advances
M2 EQUITYBITES-April 21, 2015-Oncolytics Biotech's Reolysin receives orphan drug designation from US FDA
M2 PHARMA-January 8, 2015-Gamida Cell receives orphan drug designation for NiCord from US Department of Health and Human Services