An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country.
Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development.[1] Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel.[2][3]
^Cite error: The named reference Executive was invoked but never defined (see the help page).
^Hadjivasiliou, Andreas (October 2014), "Orphan Drug Report 2014" (PDF), EvaluatePharma, retrieved 28 June 2015
^Cite error: The named reference Daly_2002 was invoked but never defined (see the help page).
An orphandrug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphandrug would not be profitable to produce...
The OrphanDrug Act of 1983 is a law passed in the United States to facilitate development of orphandrugs—drugs for rare diseases such as Huntington's...
financial incentives from governments or other agencies. Orphandrugs are medications targeting orphan diseases. Most rare diseases are genetic in origin and...
and Drug Administration. Archived from the original on 7 April 2020. Retrieved 31 May 2007. "The OrphanDrug Act (as amended)". U.S. Food and Drug Administration...
approved orphandrugs. An orphandrug may cost as much as $400,000 annually. Monopolizing orphandrugs has proven to be a very profitable strategy for drug companies;...
increased to 200. Specialty drugs may also be designated as orphandrugs or ultra-orphandrugs under the U. S. OrphanDrug Act of 1983. This was enacted...
license application Drug discovery FDA Fast Track Development Program Good Manufacturing Practice Inverse benefit law Orphandrug TOL101 John S. McInnes...
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amyloidosis in October 2018. The application for inotersen was granted orphandrug designation. It was developed by Ionis Pharmaceuticals and licensed to...
source?] The US Food and Drug Administration (FDA) granted the application for efgartigimod alfa fast track and orphandrug designations. The FDA granted...
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competitive inhibition. In 1983 the US Food and Drug Administration approved acetohydroxamic acid (AHA) as an orphandrug for "prevention of so-called struvite...
medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy)...
"Itraconazole OrphanDrug Designation". U.S. Food and Drug Administration (FDA). 16 August 2016. Retrieved 15 May 2020. "Itraconazole OrphanDrug Designation"...
Orphan Black is a Canadian science-fiction thriller television series created by screenwriter Graeme Manson and director John Fawcett and starring Tatiana...
oxybate component of Xywav was granted orphandrug designation in November 1994 by the FDA. Xywav is a prescription drug and a Schedule III controlled substance...
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"Acalabrutinib OrphanDrug Designation and Approval". U.S. Food and Drug Administration (FDA). Retrieved 15 April 2020. "Acalabrutinib OrphanDrug Designation...
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