Newron Pharmaceuticals S.p.A., a biopharmaceutical company headquartered in Bresso, Italy received Orphan Drug Designation (ODD) from the U.S. FDA for sarizotan for the treatment of Rett syndrome.  Rett Syndrome is a rare genetic disorder of the grey matter of the brain characterized with a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability.  Almost all cases of Rett syndrome are caused by a mutation in the gene encoding methyl cytosine binding protein 2 (MeCP2), which is involved in regulating gene expression important for brain development.  Currently, there is no cure for Rett syndrome and treatment is focusing on the management of symptoms.  Sarizotan is a highly selective compound for specific serotonin or dopamine receptors that modulates the activity of these neurotransmitters in the brain.  In preclinical evaluation studies, sarizotan has shown promising results on normalizing the abnormal breathing pattern in models of Rett Syndrome.  The FDA’s ODD follows the positive opinion sarizotan received for the treatment of Rett syndrome from the COMP (Committee for Orphan Medicinal Products) from the European Medicines Agency in June.

 

References:

  1. http://www.newron.com/ENG/Default.aspx?SEZ=3&PAG=141
  2. http://www.newron.com/ENG/Default.aspx?SEZ=5&PAG=19&MOD=NWRPRS