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Year : 2006  |  Volume : 38  |  Issue : 5  |  Page : 375-

Ultra orphan drugs and diseases

D Kumar, S Gupta 
 Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001, India

Correspondence Address:
D Kumar
Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001

How to cite this article:
Kumar D, Gupta S. Ultra orphan drugs and diseases.Indian J Pharmacol 2006;38:375-375

How to cite this URL:
Kumar D, Gupta S. Ultra orphan drugs and diseases. Indian J Pharmacol [serial online] 2006 [cited 2023 Jun 5 ];38:375-375
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The educational forum, 'Orphan diseases and drugs', (Indian J Pharmacol 2006; 38:171-6) is a very informative article. The various aspects of orphan diseases and drugs have been well covered. A new and interesting term, 'ultra orphan drugs and diseases', was however not included in the article. Ultra orphan drugs[1] is the term designated to drugs which are used to treat exceptionally rare and chronically debilitating or life threatening diseases. Ultra orphan disease is the term designated to diseases with a prevalence of less than 0.18 case per 10,000 population.[2] A few examples of ultra orphan drugs/diseases are[3]

Imantinib for gastrointestinal stromal tumours.Laronidase for mucopolysaccharidosis type 1.L-carnitine for genetic carnitine deficiency.Anagrelide for essential thrombocytopenia.Miglustat for Gaucher's disease.Imiglucerase for Gaucher's disease.Agalsidase for Fabry's disease.Orfadin for hereditary tyrosinaemia type 1.Sacrosidase for congenital sucrase isomaltase deficiency.


1Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005;98:829-36.
2Rawlins M. Managing rare diseases. London: Royal College of Physicians; 2004.
3McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331:1016-9.