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 ?
The legislation states that the clinical dossier of an orphan drug or vaccine should be built on a realistic assessment of the qualitative and quantitative nature of the studies that can be performed.
However, consider a different view: Bristol-Myers Squibb was shielded from the possibility that other companies would have obtained Taxol or used the Government's own clinical data on Taxol trials to file an earlier FDA application - a particularly important issue when it was thought Taxol would be protected under the Orphan Drug Act.
The United States Food and Drug Administration (FDA) has granted orphan drug designation to United States-based Stoke Therapeutics for its lead product candidate, STK-001, an investigational new treatment for Dravet syndrome, a severe and progressive genetic epilepsy, it was reported yesterday.
Taiwan biopharma company OBI Pharma Inc (TPEx:4174) reported on Wednesday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation (ODD) for OBI-3424 for the treatment of Acute Lymphoblastic Leukemia (ALL),also known as Acute Lymphocytic Leukemia.
"We see the FDA's granting of orphan drug designation for PCM-075 as underscoring the medical need for new therapies for patients with AML and an important step forward in our clinical development program."
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.
Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended.
Clavis Pharma has also applied for orphan drug status for the product in the USA as a potential new treatment for pancreatic cancer.
Once orphan designation is granted, the TGA waives evaluation fees and sets up a priority evaluation pathway for processing orphan drugs.
"Receiving EMEA orphan drug designation for decitabine may accelerate the approval process in Europe by allowing us to combine the results of our Phase III program in the United States with previous European studies that have produced positive results," said Dr.
Nord is a federation of voluntary health agencies and with rare disorders dedicated to the identification, treatment, and cure of "orphan diseases." We worked for passage of the Orphan Drug Act in 1983, and we continue to monitor the law to ensure that it lives up to its promise of promoting new treatments and cures for rare disorders.
These two biologicals/drugs have been designated as orphan drugs pursuant to the Orphan Drug Act of 1983.