Orphans no more: Pharma firms step in to tackle rare diseases


Written on Monday, November 20, 2006 by Gemini

They are orphans. No one to advocate their cause, sponsor them or give them a future. They lie in the dumpheap of the pharmaceutical industry, discarded by both the big and small pharmaceutical firms. A disease which has not been “adopted” by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications is known as an orphan disease.

According to the US Food and Drug Administration, an orphan disease is one that affects less than five patients in 10,000 inhabitants. There are dozens of experimental drugs that seem to have great potential to cure these orphan diseases. Yet the industry is reluctant to pursue them as the market isn’t profitable enough to justify the cost. So the USFDA decided to offer tax incentives so that some firms would at least give orphan drugs a once-over. Since research and development of drugs to treat such diseases is huge, the US decided to reward the firms with tax reductions and marketing exclusivity on that drug for seven years to encourage more companies to invest money in research. Similar legislations have been adopted by Europe, Australia and Japan.

Now, more than 200 orphan drugs have been approved by the USFDA and are on the market. In 2003, the leading orphan drug by worldwide sales was Amgen’s Epogen, with sales of $2.4 billion. Due to the varied sops on offer, many drugs have been developed, including those to treat glioma, multiple myeloma and cystic fibrosis. In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development and 249 orphan drugs have received marketing authorisation in the US. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.

Closer home, Ranbaxy Laboratories recently brought home an ‘orphan’ from Switzerland. The Delhi-based firm signed an agreement with Swiss biopharmaceutical firm Debiopharm to market a drug used in treatment of portal hypertension, which is an increase in the pressure within the portal vein (the vein that carries blood from the digestive organs to the liver). The new chemical entity, Sanvar (vapreotide acetate), has been granted orphan drug status in the US where it is undergoing Phase III clinical trials.

Several Switzerland-based biopharmaceutical firms are crazy to adopt. Developing drugs that treat diseases the industry has abandoned, specifically parasitic illnesses that plague the developing world, is not everyone’s cup of tea. “The granting of the orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive or say unprofitable to develop under normal circumstances,’” said Jessica Mann, medical director of Speedel, a Basel-based biopharmaceutical company, which is looking at orphan drugs.

A non profit pharmaceutical company in the US, OneWorld’s first drug was paromomycin, an injectable antibiotic, to treat visceral leishmaniasis, a deadly parasitic infection that afflicts 1.5 million people in India, and kills 200,000 people each year. The World Health Organisation had got the rights to the injectable form of paromomycin from Pharmacia - now Pfizer. It had completed mid-stage trials, but had shelved the drug due to a tight budget. OneWorld decided to go ahead with trials on its own. The WHO and OneWorld now jointly own the license to the injectable form of paromomycin, which will be available in the Indian market early next year.


  • The Europe Orphan Drug Regulation, which came into force in January 2000, defines rare diseases as an incidence of less than five patients in 10,000 inhabitants.
  • The definition, however, is not absolute as a disease can be rare in one region while being common in another. Leprosy is a rare disease in Europe but it is more prevalent in central Africa
  • List of these diseases changes over time. AIDS was once an orphan disease, but is now a high-incidence disease globally

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