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.
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, 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 ?
Biotechnology company Zealand Pharma A/S (CPH:ZEAL) announced on Tuesday the grant by the US Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) of an orphan drug designation to glepaglutide for the treatment of the rare disease short bowel syndrome (SBS).
The Orphan Drug Act also provides a seven-year exclusivity period that blocks competition and gives other financial incentives to companies that develop drugs for diseases that affect fewer than 200,000 people.
Although marketing approval with orphan drug exclusivity is targeted to the development of drugs for rare diseases or diseases in the U.
This report provides an analysis of the orphan drug market.
Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended.
a product can be designated an orphan drug if it is meant to treat an illness that affects fewer than 200,000 people.
We are gratified to have received rapid approval for this designation, which complements the orphan drug designation that decitabine received in the U.
We appreciate the thoughtful review by the FDA of our orphan drug application and believe that the incentives provided by the orphan drug law are working well to bring new health care opportunities to patients with uncommon diseases,'' said L.
The Orphan Drug Act is designed to encourage for-profit companies to invest in developing drags for less common conditions--and all 3 of the ABC drugs for MS were developed under its protections.
Pluristem Therapeutics announced that the FDA has granted the company an orphan drug designation for its PLX-R18 cell therapy for the prevention and treatment of acute radiation syndrome, or ARS.
M2 EQUITYBITES-June 12, 2017-Wilson Therapeutics announces US Orphan Drug Designation receipt for WTX101 to treat ALS
M2 PHARMA-November 18, 2016-Amplyx Pharmaceuticals' APX001 receives USFDA orphan drug designation