PHARMACEUTICAL COMPOSITION OF THE GLUCOSYLCERAMIDE SYNTHASE INHIBITOR ELIGLUSTAT FOR THE TREATMENT OF GAUCHER'S DISEASE COMPRISING ADJUSTING THE INDIVIDUAL THERAPEUTICAL DOSE TO THE P450 METABOLISM OF THE PATIENT

摘要

The present invention relates to a pharmaceutical composition comprising the hemitartrate salt of Eliglustat (Genz 112638; N-[(1R,2R)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]-octanamide) represented by the following structural formula:(Formula I Hemitartrate)as an inhibitor of the glucosylceramide synthase (GCS) for use in a method of treating Fabry's or Gaucher's disease, the method comprising adjusting the individual therapeutical dose to the P450 metabolism of the patient.Particular single crystalline forms of Eliglustat Hemitartrate are characterized by a variety of properties and physical measurements as well as methods of producing crystalline Eliglustat Hemitartrate and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations 10 in subjects to treat Fabry's or Gaucher's disease, are also discussed.