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Drug Repurposing and Repositioning Workshop Summary (2014) / Chapter Skim
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4 Value Propositions for Drug Repurposing
Pages 31-38

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From page 31...
... In a market where drug development is costly, pharmaceutical companies carefully consider their strategies for which drugs and targets to pursue, individual speakers said. The repurposing of thalidomide is provided as an example of the importance of understanding drug and disease mechanisms coupled with clinical data.
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From page 32...
... NOTE: FDA, U.S. Food and Drug Administration; HIV, human immunodeficiency virus; PDUFA, Prescription Drug User Fee Act; R&D, research and development; US, United States.
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From page 33...
... Gaining a better understanding of disease targets and mechanisms can overcome market imperfections that impede repurposing. Some companies are developing platform tools to build matrices of diseases and molecules that might treat those diseases.
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From page 34...
... REPURPOSING DRUGS CURRENTLY IN DEVELOPMENT Translating scientific insights into novel therapies requires identifying the area where scientific innovation, unmet needs, and commercial attractiveness overlap, observed Simeon Taylor, vice president for research and scientific affairs at Bristol-Myers Squibb (BMS)
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From page 35...
... Another example involves lomitapide, which was recently approved as a treatment for familial hypercholesterolemia. The basic scientific research conducted largely at BMS relied on animal models and found that the compound effectively improved lipid profiles, Taylor said.
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From page 36...
... THALIDOMIDE: REPURPOSING A DRUG THAT WAS NOT SUCCESSFUL FOR ITS FIRST INDICATION Thalidomide was initially marketed as a sedative to address symptoms of morning sickness in pregnant women and was first offered as an over-the-counter product in 1957 by the German pharmaceutical company 1 On February 24, 2014, FDA approved Myalept (metreleptin) for patients with congenital generalized or acquired generalized lipodystrophy.
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From page 37...
... . In the United States, FDA reviewer Frances Oldham Kelsey had delayed approval of the drug until a more complete data package was made available, which saved many U.S.
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From page 38...
... For example, a molecule called CC-122 is in late Phase I studies showing dramatic activity in B cell malignancies, in hepatocellular carcinomas, and in anaplastic astrocytoma, Daniel said. Data Coupling Is Key An understanding of disease mechanisms and therapeutic targets can dramatically narrow the hunt for clinical indications and provide paths for regulatory approval, Daniel said; however, clinical observations remain important for drug repurposing, as demonstrated by the use of thalidomide for leprosy and other conditions.
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Key Terms

  • drug development
  • value propositions
  • disease mechanisms
  • regulatory approval
  • drug indications

  • biotechnology company
  • clinical data
  • effective treatments
  • therapeutic targets
  • related compounds

  • government incentives
  • adverse reactions
  • familial hypercholesterolemia
  • prescription drug
  • drug user

  • particular drug
  • incentivizes drug
  • drug failed
  • understanding drug
  • stop drug

  • patients receive
  • disease targets
  • metabolic disease
  • sufficient endogenous
  • separate biotechnology

  • repurposing drugs
  • bms program
  • insights linking
  • treat patients
  • indication thalidomide


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