US2004248793A1PendingUtilityA1

Stabilised solid compositions of factor VII polypeptides

Priority: Jun 21, 2002Filed: Jun 30, 2003Published: Dec 9, 2004
Est. expiryJun 21, 2022(expired)· nominal 20-yr term from priority
A61P 7/02A61P 7/04A61P 7/00A61K 47/20C12Y 304/21021A61K 38/4846A61K 9/0019A61K 47/26A61K 47/22A61K 47/02A61K 9/19A61K 47/08A61K 47/10A61K 47/34A61K 38/36A61K 47/183
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Claims

Abstract

The invention relates to chemically as well as physically stable compositions comprising Factor VII or a Factor VII-related polypeptide such that these compositions can be stored, handled and used at room temperature.

Claims

exact text as granted — not AI-modified
1 . A composition comprising a Factor VII polypeptide, and at least one stability agent selected from the group consisting of 
 a) a combination of an antioxidant and mannitol;    b) a combination of methionine and a polyol;    c) a combination of a saccharide and mannitol;    d) a combination of sucrose and a polyol; and    e) methionine,    said composition having a moisture content of not more than about 3%.    
     
     
         2 . The composition according to  claim 1 , wherein the composition comprises a combination of an antioxidant and mannitol and further comprises a saccharide.  
     
     
         3 . The composition according to  claim 1 , wherein the composition comprises a combination of methionine and a polyol and further comprises a saccharide.  
     
     
         4 . The composition according to  claim 1 , wherein the composition comprises a combination of a saccharide and mannitol and further comprises an antioxidant.  
     
     
         5 . The composition according to  claim 1 , wherein the composition comprises a combination of sucrose and a polyol and further comprises an antioxidant.  
     
     
         6 . The composition according to  claim 1 , wherein the antioxidant is selected from the group consisting of homocysteine, cysteine, cystathionine, methionine, gluthatione, and peptides containing any one of homocysteine, cysteine, cystathionine, methionine, and gluthatione.  
     
     
         7 . The composition according to  claim 1 , wherein the saccharide is selected from the group consisting of sucrose, dextrose, lactose, maltose, trehalose, cyclodextrins, maltodextrins and dextrans.  
     
     
         8 . The composition according to  claim 1 , wherein the polyol is selected from the group consisting of mannitol, sorbitol and xylitol.  
     
     
         9 . The composition according to  claim 1 , wherein the composition is stable such that not more than about 5% w/w of the initial content of Factor VII polypeptide is converted to aggregates upon storage of said composition at 30° C. for 8 months.  
     
     
         10 . The composition according to  claim 1 , wherein the composition is stable such that not more than about 6% w/w of the initial content of Factor VII polypeptide is converted to oxidised forms upon storage of said composition at 30° C. for 8 months.  
     
     
         11 . The composition according to  claim 1 , wherein said polyol is in an amount ranging from about 5% w/w to 90% w/w.  
     
     
         12 . The composition according to  claim 1 , wherein said saccharide is in an amount ranging from about 0 to 85% w/w.  
     
     
         13 . The composition according to  claim 1 , wherein said polyol is in a weight ratio relative to said saccharide ranging from about 100:1 to 1:50.  
     
     
         14 . The composition according to  claim 1 , further comprising an agent suitable for keeping the pH of said composition in the range of 3 to 9 when dissolved in aqueous solvent.  
     
     
         15 . The composition according to  claim 14 , wherein said agent is selected from the group consisting of citrate, acetate, histidine, malate, phosphate, tartaric acid, succinic acid, MES, HEPES, PIPES, imidazol, TRIS, lactate, glutamate and glycylglycine.  
     
     
         16 . The composition according to  claim 1 , further comprising a tonicity modifier.  
     
     
         17 . The composition according to  claim 16 , wherein the tonicity modifier is selected from the group consisting of sodium acetate, sodium lactate, sodium chloride, potassium chloride and calcium chloride.  
     
     
         18 . The composition according to  claim 1 , further comprising a surfactant.  
     
     
         19 . The composition according to  claim 18 , wherein the surfactant is selected from the group consisting of polysorbates; polyoxyethylene alkyl ethers; poloxamers; ethylene/polypropylene block polymers; and polyethyleneglycols (PEGs).  
     
     
         20 . The composition according to  claim 1 , further comprising one or more other pharmaceutical excipients acting as a bulking agent.  
     
     
         21 . The composition according to  claim 1 , wherein the saccharide is sucrose.  
     
     
         22 . The composition according to  claim 1 , wherein the polyol is mannitol.  
     
     
         23 . The composition according to  claim 1 , wherein the Factor VII Polypeptide is selected from the group consisting of Human Factor VIIa and a Factor VII Sequence Variant.  
     
     
         24 . The composition according to  claim 23 , wherein the Factor VII Polypeptide is recombinant human Factor VIIa.  
     
     
         25 . The composition according to  claim 1 , wherein the Factor VII Polypeptide is a Factor VII-related polypeptide and wherein the ratio between the activity of said Factor VII-related polypeptide and wild-type Factor VII is at least 1.25 when tested in an in vitro proteolysis assay or in an in vitro hydrolysis assay.  
     
     
         26 . The composition according  claim 23 , wherein Factor VII polypeptide is present in a concentration of from about 0.6 mg/ml to about 10.0 mg/ml.  
     
     
         27 . The composition according to  claim 1 , wherein said moisture content is not more than about 2.5% w/w.  
     
     
         28 . The composition according to  claim 1 , wherein the composition is a lyophilised cake.  
     
     
         29 . The composition according to  claim 1 , wherein the composition comprises: Factor VII polypeptide, Mannitol, Sucrose, and a surfactant selected from the group consisting of a polysorbate and a poloxamer.  
     
     
         30 . The composition according to  claim 29 , further comprising methionine.  
     
     
         31 . The composition according to  claim 29 , further comprising L-histidine.  
     
     
         32 . The composition according to  claim 29 , further comprising one or more components selected from the group consisting of: CaCl 2 , NaCl, and Glycylglycine.  
     
     
         33 . The composition according to  claim 1 , wherein the composition is selected from the group consisting of formulations A, B, C, and D:  
       
         
           
                 
                 
                 
                 
                 
               
                     
                 
                     
                 
                   Compound 
                   Formulation A 
                   Formulation B 
                   Formulation C 
                   Formulation D 
                 
                     
                 
                   FVII polypeptide 
                   0.6 to 10 mg/ml 
                   0.6 to 10 mg/ml 
                   0.6 to 10 mg/ml 
                   0.6 to 10 mg/ml 
                 
                   CaCl2 x2H2O 
                   5 to 20 mM 
                   5 to 20 mM 
                   5 to 20 mM 
                   5 to 20 mM 
                 
                   NaCl 
                   0-50 mM 
                   0-50 mM 
                   0-50 mM 
                   0-50 mM 
                 
                   Glycylglycine 
                   0-15 mM 
                   0-15 mM 
                   0-15 mM 
                   0-15 mM 
                 
                   L-Histidine 
                   0-20 mM 
                   0-20 mM 
                   0-20 mM 
                   0-20 mM 
                 
                   Mannitol 
                   20 to 40 mg/ml 
                   20 to 40 mg/ml 
                   20 to 40 mg/ml 
                   20 to 40 mg/ml 
                 
                   Sucrose 
                   5 to 20 mg/ml 
                   — 
                   — 
                   5 to 20 mg/ml 
                 
                   Methionine 
                   0-1 mg/ml 
                   0-1 mg/ml 
                   0-1 mg/ml 
                   0-1 mg/ml 
                 
                   Tween 80 
                   0.05 to 0.15 mg/ml 
                   0.05 to 0.15 mg/ml 
                 
                   Poloxamer 188 
                   — 
                   — 
                   0.5-3 mg/ml 
                   0.5-3 mg/ml 
                 
                   Ph 
                   5.0 to 7.0 
                   5.0 to 7.0 
                   5.0 to 7.0 
                   5.0 to 7.0 
                 
                     
                 
                     
                 
             
                
                
                
                
               
               
                
                
                
                
                
                
                
                
                
                
                
                
                
               
            
           
         
       
     
     
         34 . The composition according to  claim 1 , wherein the composition is selected from the group consisting of formulations Ex, Fx, Gx, Hx, Ix, Jx, Kx, and Lx:  
       
         
           
                 
                 
                 
                 
                 
               
                     
                 
                     
                 
                   Compound 
                   Formulation Ex 
                   Formulation Fx 
                   Formulation Gx 
                   Formulation Hx 
                 
                     
                 
                   FVIIa polypeptide 
                   1.0 mg/ml 
                   1.0 mg/ml 
                   1.0 mg/ml 
                   1.0 mg/ml 
                 
                   CaCl2 x2H2O 
                   10 mM 
                   10 mM 
                   10 mM 
                   10 mM 
                 
                   NaCl 
                   39 mM 
                   39 mM 
                   39 mM 
                   39 mM 
                 
                   Glycylglycine 
                   10 mM 
                   10 mM 
                   10 mM 
                   10 mM 
                 
                   Mannitol 
                   25 mg/ml 
                   25 mg/ml 
                   25 mg/ml 
                   25 mg/ml 
                 
                   Sucrose 
                   10 mg/ml 
                   10 mg/ml 
                   10 mg/ml 
                   10 mg/ml 
                 
                   Methionine 
                   0.5 mg/ml 
                   0.5 mg/ml 
                   0.5 mg/ml 
                   0.5 mg/ml 
                 
                   Tween 80 
                   0.1 mg/ml 
                   — 
                   0.1 mg/ml 
                   — 
                 
                   Poloxamer 188 
                   — 
                   1.0 mg/ml 
                     
                   1.0 mg/ml 
                 
                   L-Histidine 
                   — 
                   — 
                   10 mM 
                   10 mM 
                 
                   pH 
                   5.5 to 6.0 
                   5.5 to 6.0 
                   5.5 to 6.0 
                   5.5 to 6.0 
                 
                     
                 
                   Compound 
                   Formulation Ix 
                   Formulation Jx 
                   Formulation Kx 
                   Formulation Lx 
                 
                     
                 
                   FVII polypeptide 
                   1.0 mg/ml 
                   1.0 mg/ml 
                   1.0 mg/ml 
                   1.0 mg/ml 
                 
                   CaCl2 x2H2O 
                   10 mM 
                   10 mM 
                   10 mM 
                   10 mM 
                 
                   NaCl 
                   39 mM 
                   39 mM 
                   39 mM 
                   39 mM 
                 
                   Glycylglycine 
                   10 mM 
                   10 mM 
                   10 mM 
                   10 mM 
                 
                   Mannitol 
                   25 mg/ml 
                   25 mg/ml 
                   25 mg/ml 
                   25 mg/ml 
                 
                   Sucrose 
                   10 mg/ml 
                   10 mg/ml 
                   10 mg/ml 
                   10 mg/ml 
                 
                   Methionine 
                   — 
                   — 
                   — 
                   — 
                 
                   Tween 80 
                   0.1 mg/ml 
                   — 
                   0.1 mg/ml 
                   — 
                 
                   Poloxamer 188 
                   — 
                   1.0 mg/ml 
                     
                   1.0 mg/ml 
                 
                   L-Histidine 
                   — 
                   — 
                   10 mM 
                   10 mM 
                 
                   pH 
                   5.5 to 6.0 
                   5.5 to 6.0 
                   5.5 to 6.0 
                   5.5 to 6.0 
                 
                     
                 
                     
                 
             
                
                
               
               
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
               
            
           
         
       
     
     
         35 . The compositions according to  claim 29 , wherein the Factor VII polypeptide is human Factor VIIa.  
     
     
         36 . A method of preparing a stable Factor VII polypeptide comprising the steps of: 
 i) providing said Factor VII polypeptide in a solution comprising at least one stability agent selected from the group consisting of    a) a combination of an antioxidant and mannitol;    b) a combination of methionine and a polyol;    c) a combination of a saccharide and mannitol;    d) a combination of sucrose and a polyol; and    e) methionine; and    ii) processing said solution so as to obtain a solid composition with a moisture content not more than about 3% wiw.    
     
     
         37 . The method according to  claim 36 , wherein the antioxidant is selected from the group consisting of homocysteine, cysteine, cystathionine, methionine, gluthatione, and peptides containing any one of homocysteine, cysteine, cystathionine, methionine and gluthatione.  
     
     
         38 . The method according to  claim 36 , wherein the saccharide is selected from the group consisting of sucrose, dextrose, lactose, maltose, trehalose, cyclodextrins, maltodextrins and dextrans.  
     
     
         39 . The method according to  claim 36 , wherein the polyol is selected from the group consisting of mannitol, sorbitol and xylitol.  
     
     
         40 . The method according to  claim 36 , wherein said polyol is present in an amount ranging from about 0.5 to about 75 mg/ml.  
     
     
         41 . The method according to  claim 36 , wherein said saccharide is present in an amount ranging from about 0.5 to 75 mg/ml.  
     
     
         42 . The method according to  claim 36 , wherein said antioxidant is in an amount ranging from about 0.05 to 10 mg/ml.  
     
     
         43 . The method according to  claim 36 , wherein the saccharide is sucrose.  
     
     
         44 . The method according to  claim 36 ,  43 , wherein the antioxidant is methionine.  
     
     
         45 . The method according to  claim 36 , wherein the polyol is mannitol.  
     
     
         46 . The method according to  claim 36 , wherein said processing comprises freeze-drying.  
     
     
         47 . A method for treating a Factor VII-responsive syndrome, said method comprising administering to a subject in need thereof an effective amount of a composition comprising a Factor VII polypeptide, and at least one stability agent selected from the group consisting of 
 a) a combination of an antioxidant and mannitol;    b) a combination of methionine and a polyol;    c) a combination of a saccharide and mannitol;    d) a combination of sucrose and a polyol; and    e) methionine;    said composition having a moisture content not more than about 3%.    
     
     
         48 . The method according to  claim 47 , wherein said syndrome is selected from the group consisting of haemophilia A, haemophilia B, Factor XI deficiency, Factor VII deficiency, thrombocytopenia, von Willebrand's disease, presence of a clotting factor inhibitor, surgery, trauma, dilutional coagulopathy, and anticoagulant therapy.  
     
     
         49 . The method according to  claim 47 , further comprising the step of dissolving the composition in a suitable liquid prior to the administering step.

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