A drug to cure cutaneous neurofibroma could be worth $3B

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According to the NY Times and NPR, drugs for orphan diseases have become big business, partly due to side-effects of a law intended to promote development.  The Times discusses two recent sales:

  1. $3.3 billion for rights to the drug Kalydeco for cystic fibrosis, which has a frequency of 1:3500 births.
  2. $2.85 billion for rights to the drug Tysabri for multiple sclerosis, which has a frequency of 1:750.

Cutaneous neurofibroma is a kind of benign skin tumor suffered by most people with NF1, which means that there is a mutation in the gene which codes for neurofibromin 1, which is a tumor suppressing protein.  NF1 has a frequency of 1:3000 which makes it more frequent than CF and less frequent than MS.  By the numbers above, a drug which prevents or eliminates cutaneous neurofibroma would be worth $3billion.

The drug selumetinib was discovered by Array BioPharma and licensed to  AstroZeneca.  The agreement has had some difficulties.  Selumetinib was approved to treat neurofibroma.  An early phase trial shows tumor reduction but not elimination.   Selumetinib is an MEK inhibitor.

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