Methods for treating lysosomal acid lipase deficiency in patients
AssigneeAlexion Pharmaceuticals, Inc.
InventorAnthony QUINN
Life of this patent
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Abstract
The present invention provides methods of treating LAL deficiency comprising administering to a mammal a therapeutically effective amount of lysosomal acid lipase with an effective dosage frequency. Methods of improving growth and liver function, increasing LAL tissue concentration, and increasing LAL activity in a human patient suffering from LAL deficiency are also provided.
Claims16
(canceled)
A glass vial comprising a pharmaceutical composition comprising:
- (a) 2 mg/ml of human recombinant lysosomal acid lipase (LAL);
- (b) 13.7 mg/ml of trisodium citrate dehydrate;
- (c) 1.57 mg/ml of citric acid monohydrate;
- and
- (d) 10 mg/ml of human serum albumin,
- wherein the pharmaceutical composition comprises a total volume of 10 mL or 10.5 mL and a pH of 5.9±0.2.
A method for determining a number of single-use glass vials containing recombinant human LAL needed for treating a human patient suffering from an LAL deficiency, the method comprising the steps of:
- (a) selecting a dose for a subject of 1 mg/kg, 3 mg/kg, or 5 mg/kg
- (b) obtaining the subject's weight in kg;
- (c) calculating a total dose number (mg) by multiplying the subject's weight (kg) by the selected dose (mg/kg);
- (d) calculating a total vial number by dividing the total dose number by the number of mg per vial;
- and
- (e) rounding the total vial number up to the nearest integer,
- wherein the integer is the total number of single-use glass vials needed for treating a human patient suffering from the LAL deficiency.
A method of treating a human subject suffering from a LAL deficiency, comprising administering an escalated dose of 3 mg/kg of a recombinant human LAL to the subject, wherein the subject has been determined to have a suboptimal clinical response following administration of 1 mg/kg of the recombinant human LAL.
A method of treating a human subject suffering from a LAL deficiency, comprising administering to the subject 1 mg/kg of recombinant human LAL once every 7 or 14 days.
A method of treating a human subject suffering from a LAL deficiency, comprising administering to the subject 5 mg/kg of recombinant human LAL between one time every 5 days and one time every 30 days.
Dependencies are inferred from the claim text.