Methods for treating gaucher disease

Abstract

Methods for treating Gaucher disease in patients with renal or hepatic impairment.

1 . A method of treating Gaucher disease comprising administering to a patient in need thereof an effective amount of eliglustat, or a pharmaceutically acceptable salt thereof, wherein said patient is an extensive CYP2D6 metabolizer with mild hepatic impairment. 
     
     
         2 . The method according to  claim 1 , wherein the effective amount is a twice daily dose of 84 mg of eliglustat, or a pharmaceutically acceptable salt thereof, measured in base form. 
     
     
         3 . The method according to  claim 2  wherein the eliglustat or pharmaceutically acceptable salt thereof is eliglustat hemitartrate. 
     
     
         4 . A method of treating Gaucher disease comprising administering to a patient in need thereof an adjusted effective amount of eliglustat, or a pharmaceutically acceptable salt thereof, wherein said patient is an extensive CYP2D6 metabolizer with mild hepatic impairment and wherein said patient is concurrently taking a drug that is a weak CYP2D6 inhibitor. 
     
     
         5 . The method according to  claim 4  wherein the adjusted effective amount is a once daily dose of 84 mg of eliglustat or a pharmaceutically acceptable salt thereof, measured in base form. 
     
     
         6 . The method according to  claim 5 , wherein the eliglustat or pharmaceutically acceptable salt thereof is eliglustat hemitartrate. 
     
     
         7 . A method of treating Gaucher disease comprising administering to a patient in need thereof an adjusted effective amount of eliglustat, or a pharmaceutically acceptable salt thereof, wherein said patient is an extensive CYP2D6 metabolizer with mild hepatic impairment and wherein said patient is concurrently taking a drug that is a weak CYP3A inhibitor. 
     
     
         8 . The method according to  claim 7 , wherein the adjusted effective amount is a once daily dose of 84 mg of eliglustat or a pharmaceutically acceptable salt thereof, measured in base form. 
     
     
         9 . The method according to  claim 8 , wherein the eliglustat or pharmaceutically acceptable salt thereof is eliglustat hemitartrate. 
     
     
         10 .- 12 . (canceled) 
     
     
         13 . A method of providing eliglustat, or a pharmaceutically acceptable salt thereof, wherein the eliglustat, or pharmaceutically acceptable salt thereof, is provided along with information indicating that it is useful for treating patients with Gaucher disease, and in cases wherein said patient is an extensive CYP2D6 metabolizer with mild hepatic impairment, no dose adjustment is required. 
     
     
         14 . An article of manufacture comprising
 a) a packaging material;   b) eliglustat, or a pharmaceutically acceptable salt thereof; and   c) a label or package insert contained within the packaging material indicating that eliglustat, or a pharmaceutically acceptable salt thereof, is contraindicated in CYP2D6 intermediate metabolizers (IMs) or poor metabolizers (PMs) with any degree of hepatic impairment and in CYP2D6 extensive metabolizers (EMs) with moderate or severe hepatic impairment.   
     
     
         15 . A package comprising eliglustat, or a pharmaceutically acceptable salt thereof, and a label, said label comprising a printed statement which informs a prospective user that eliglustat, or pharmaceutically acceptable salt thereof, is i) indicated for the treatment of Gaucher disease type 1 and ii) is contraindicated in CYP2D6 intermediate metabolizers (IMs) or poor metabolizers (PMs) with any degree of hepatic impairment and in CYP2D6 extensive metabolizers (EMs) with moderate or severe hepatic impairment. 
     
     
         16 . A method for treating Gaucher disease, comprising administering an adjusted effective amount of eliglustat, or a pharmaceutically acceptable salt thereof, to a patient in need thereof in combination with a weak CYP2D6 inhibitor, wherein said patient is a CYP2D6 extensive metabolizer and wherein said patient has mild hepatic impairment. 
     
     
         17 . The method according to  claim 16  wherein the adjusted effect amount of eliglustat, or pharmaceutically acceptable salt thereof, is a dose of 84 mg of eliglustat, measured in base form, once daily. 
     
     
         18 . The method according to  claim 17 , wherein the eliglustat or pharmaceutically acceptable salt thereof is eliglustat hemitartrate. 
     
     
         19 . A method for treating Gaucher disease, comprising administering an adjusted effective dose of eliglustat, or a pharmaceutically acceptable salt thereof, to a patient in need thereof in combination with a weak CYP3A inhibitor, wherein said patient is a CYP2D6 extensive metabolizer and wherein said patient has mild hepatic impairment. 
     
     
         20 . The method according to  claim 19 , wherein the adjusted effect amount of eliglustat, or pharmaceutically acceptable salt thereof, is a dose of 84 mg of eliglustat, measured in base form, once daily. 
     
     
         21 . The method according to  claim 20 , wherein the eliglustat or pharmaceutically acceptable salt thereof is eliglustat hemitartrate.