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.

Mentioned in: Down Syndrome, Huntington Disease, Polycythemia Vera, Severe Combined Immunodeficiency

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References in periodicals archive

The role of patient groups in increasing awareness of orphan drug development has been widely recognized for pharmaceutical orphan drugs in the United States and has been emphasized in the European project.

Development of orphan vaccines: an industry perspective

One example of the financial implications of the Orphan Drug Act is Celgene, which has been one of the top beneficiaries of the legislation.

Concerns over orphan drug costs overblown

Furthermore, it is important to recognize that the nutritional supplement problem is not a rare disease problem, and it therefore has no relationship to the Orphan Drug Act.

The Irony of Nutritional Supplements

Orphan Drug Designation in Europe is offered to companies developing products aimed at treating a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 persons in the European Union.

Aridis Pharmaceuticals' AR-501 receives Orphan Drug Designation

"Receiving orphan drug designation is an important regulatory milestone," said Steve Lisi, chairman and chief executive officer of AIT Therapeutics.

AIT Therapeutics Inc. was granted orphan drug designation by the FDA for its novel Nitric oxide (NO)

"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.

Report assesses FDA's handling of orphan drugs

14 September 2009 - The European Medicines Agency (EMEA) has recommended granting orphan drug status to the Intravenous CP-4126 drug for the treatment of pancreatic cancer of Norwegian company Clavis Pharma ASA (OSL:CLAVIS).

Norwegian Clavis Pharma's cancer drug recommended for orphan drug designation

Orphan Drug designation in the European Union provides a 10-year period of post-approval marketing exclusivity and other financial incentives for new drugs that offer significant therapeutic advantage over other approved treatments of rare conditions (conditions affecting no more than 5 in 10,000 persons).

BRAIN CANCER DRUG GETS ORPHAN DRUG DESIGNATION FROM EMEA

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." The Act established, among other things, exclusive marketing rights on unpatentable orphan drugs for a period of seven years and a tax credit equal to 50 percent of the cost of conducting human clinical trials.

Orphan Drug Act on congressional agenda

M2 EQUITYBITES-June 7, 2019-US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl

US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl

M2 PHARMA-June 7, 2019-US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl

US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl

Since its inception, the application process for orphan drug designation in the U.S.

Developments in orphan drug exclusivity after Depomed: a look at FDA's reaction to and future implications of the recent court decision in Depomed vs. U.S. Dept. of Health and Human Services