orphan drug

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orphan drug,

drug developed under the U.S. Orphan Drug Act (1983) to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax breaks and a seven-year monopoly on drug sales to induce companies to undertake the development and manufacturing of such drugs, which otherwise might not be profitable because of the small potential market. (Of the 5,000 diseases covered under the act, 47% affect fewer than 25,000 people.) The law has led to the introduction of valuable new drugs for the treatment of rare diseases, but some drug companies have been accused of abusing the law's provisions by making inordinately high profits on orphan drugs under monopoly. Since the 1983 act went into effect, many orphan drugs have been approved, including those for the treatment of such conditions as AIDSAIDS
or acquired immunodeficiency syndrome,
fatal disease caused by a rapidly mutating retrovirus that attacks the immune system and leaves the victim vulnerable to infections, malignancies, and neurological disorders. It was first recognized as a disease in 1981.
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, cystic fibrosiscystic fibrosis
, inherited disorder of the exocrine glands (see gland), affecting children and young people; median survival is 25 years in females and 30 years in males.
..... Click the link for more information.
, blepharospasm (uncontrolled rapid blinking), and snake bite.

orphan drug

[¦ȯr·fən ′drəg]
(pharmacology)
A pharmaceutical developed to treat a disease that afflicts relatively few people.
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References in periodicals archive ?
The FDA Office of Orphan Products Development manages a grant program to help drug development for rare conditions.
On the other hand, the drawback of basing a clinical dossier on a limited amount of data is the obvious difficulty in evaluating the safety profile of an orphan product with sufficient statistical confidence.
Senators Edward Kennedy (D-Mass.) and Orrin Hatch (R-Utah) claim that manufacturers are abusing the provisions by charging high prices for their orphan products. They have asked that the FDA impose a moratorium on orphan approvals until June 30, 1989, so that Congress can review the Act and consider amendments.
1379 will fortify the ORD by making it a permanent office at NIH; giving it statutory authority which will allow it to form its own research agenda; doubling its budget to $4 million in Fiscal Year 2002 and "such sums as may be necessary" for each subsequent fiscal year; authorizing new funds for Rare Disease Research Centers of Excellence in the amount of $20 million; and doubling the funding of the Orphan Product Research Grant Program at the FDA-which receives 100 grant applications yearly-to $25 million in FY 2002.
Rao, M.D., J.D., director of FDAs Office of Orphan Product Development, within the Office of Special Medical Programs.
Aastrom Biosciences, Inc., Ann Arbor, MI announced the FDA has granted orphan product status to the company's SC-1 autologous bone marrow stem cells using its AastromReplicell System.
The legislation would give statutory authority to the existing Office of Rare Diseases at NIH and increase federal funding for the ORD and for the FDA's Orphan Product Research Grants program.
Chemet is a designated orphan drug supported by the FDA's Orphan Product Development Program.
Spark Therapeutics has received orphan product designation for Luxturna from EMA for the treatment of inherited retinal dystrophies.
for VYXEOS, this acquisition would add a new orphan product with the potential for short- and long-term revenue generation and expansion of our international commercial platform."
And it held an exchange of views with Dr Marlene Haffner, Director of the Office for Orphan Product Development at the US Food and Drug Administration, which has 17 years of experience with orphan drugs.
The FDA granted Vonvendi orphan product designation for these uses.