Here’s What’s on The Orphan Drug Hall of Fame


Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases.

They respond to a public health need, but not necessarily an economic need by the pharmaceutical industry because they are used in diseases that occur so infrequently, so there is no reasonable expectation that development costs will be recovered from potential sales.

Being granted an orphan drug designation by the government allows a pharmaceutical company to various incentives and legal status, which in turn re-incentivizes the research and development of these drugs. To learn more about orphan drugs from Orphanet, visit this page!

Paul Melmeyer is the director of federal policy at the National Organization for Rare Disorders in Washington, D.C., which helped lobbied for the passage of the Orphan Drug Act in 1983. On the wall of his office are posters that mark which drugs he has designated to The Orphan Drug Hall of Fame for being exceptionally successful against rare diseases.

Here are the top three orphan drugs that made the cut and a little bit about them:

  1. Norditropin®: an injectable man-made growth hormone for certain growth hormone-related disorders/deficiencies. Norditropin was developed by Novo Nordisk and approved in August, 2006.
  2. REVLIMID®: used to treat multiple myeloma, myelodysplastic syndromes, and mantle cell lymphoma. REVLIMID® was developed by Celgene and approved in December of 2005.
  3. CYSTADANE®: an oral powder (that only actively contains betaine anhydrous) to treat homocystinuria. CYSTADANE® was made by Recordati Rare Diseases, Inc. and approved in May of 1994.

If you have been following Patient Worthy for a while, you know that every day, we are reporting on the latest clinical trials and research coming out for rare disease treatment. Maybe, some of these medications currently in development will soon join these other drugs on The Orphan Drug Hall of Fame.

To learn more about Paul Melmeyer and the work that he does, click here!




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