Methods for treating hypophosphatasia in children
AssigneeAlexion Pharmaceuticals, Inc.
InventorsKenji FUJITA, Dawn PHILLIPS
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Abstract
The disclosure features methods for treating hypophosphatasia (HPP) in a patient (e.g., a child having HPP) exhibiting physical impairments, disability in activities of daily living (ADL), pain, and/or delayed motor development by administering a soluble alkaline phosphatase (sALP) to the patient.
Claims98
A method of treating hypophosphatasia (HPP) in a child of about 5 to about 12 years of age having an average Bruininks-Oseretsky Test of Motor Proficiency 2nd Edition (BOT-2) strength score of less than about 10, wherein the method comprises administering a soluble alkaline phosphatase (sALP) to the child at a dosage providing about 6 mg/kg/week of the sALP, wherein the sALP comprises an amino acid sequence having at least 95% sequence identity to the amino acid sequence of SEQ ID NO:
- 1, and wherein administration of the sALP results in an average increase in the BOT-2 strength score to about 10 up to about 20 after a period of at least four years.
A method of treating HPP in a child of about 5 to about 12 years of age having an average BOT-2 running speed and agility score of less than 5, wherein the method comprises administering a sALP to the child at a dosage providing about 6 mg/kg/week of the sALP, wherein the sALP comprises an amino acid sequence having at least 95% sequence identity to the amino acid sequence of SEQ ID NO:
- 1, and wherein administration of the sALP results in an average increase in the BOT-2 running speed and agility score to about 5 up to about 20 after a period of at least four years.
A method of treating HPP in a child of about 5 to about 12 years of age having an average Childhood Health Assessment Questionnaire (CHAQ) disability index and/or discomfort score of greater than about 0.8, wherein the method comprises administering a sALP to the child at a dosage providing about 6 mg/kg/week of the sALP, wherein the sALP comprises an amino acid sequence having at least 95% sequence identity to the amino acid sequence of SEQ ID NO:
- 1, and wherein administration of the sALP results in an average decrease in the CHAQ disability index score and/or discomfort score to about 0 up to equal to or less than about 0.5 after a period of at least four years.
A method of treating HPP in a child of about 5 to about 12 years of age having an average Pediatric Outcomes Data Collection Instrument (PODCI) transfer and mobility, sports and physical functioning, and/or pain score of less than about 40, wherein the method comprises administering a sALP to the child at a dosage providing about 6 mg/kg/week of the sALP, wherein the sALP comprises an amino acid sequence having at least 95% sequence identity to the amino acid sequence of SEQ ID NO:
- 1, and wherein administration of the sALP results in an average increase in the PODCI score to about 40 up to about 50 after a period of at least four years.
A method of treating HPP in a child of about 5 to about 12 years of age, wherein the method comprises administering a sALP to the child at a dosage providing about 6 mg/kg/week of the sALP, wherein the sALP comprises an amino acid sequence having at least 95% sequence identity to the amino acid sequence of SEQ ID NO:
- 1, and wherein administration of the sALP for a treatment period of at least one year results in an average increase in a Six Minute Walk Test (6MWT) distance of the child relative to the 6MWT distance of the child prior to administration of the sALP.
Dependencies are inferred from the claim text.