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. Author manuscript; available in PMC: 2019 Jan 19.
Published in final edited form as: Nat Rev Drug Discov. 2018 Mar 23;17(5):317–332. doi: 10.1038/nrd.2018.14

Table 4 |.

Examples of successful attempts of targeting the dark genome

Gene name Relevant study (year) Study type and reference Citation counta API name Therapeutic indication Market approval (year)
LEPR 1995 Receptor deorphanization99 4,100 Metreleptin Lipodystrophy 2014
SMO 1998 Protein-diseaseassociation study100 1,195 Vismodegib Basal cell carcinoma 2012
S1PR1 1998 Receptordeorphanization101 968 Fingolimod Multiple sclerosis 2010
HCRTR1 and HCRTR2 1998 Receptor deorphanization102 4,608 Suvorexant Insomnia 2014
PCSK9 2003 Protein-diseaseassociation study103 1,840 Evolocumab Hypercholesterolaemia 2015
GHSR 1999 Receptor deorphanization104 8,248 Anamorelinb Cachexia Successful phase III clinical trial105

API, active pharmaceutical ingredient; GHSR, ghrelin receptor; HCTR1, orexin receptor type 1; LEPR, leptin receptor; PCSK9, proprotein convertase subtilisin/kexin type 9; S1PR1, sphingosine 1-phosphate receptor 1; SMO, smoothened homologue.

a

Citation count for the 'relevant study' reference, according to Google Scholar, as of 28 December 2017.

b

The European Medicines Agency refused marketing authorization for anamorelin in September 2017.